A few months ago, my medical school invited the first person treated for sickle cell disease with this technology to lecture about their experience, and I had the opportunity to meet them. Indeed, we live in exciting times!
I am a pre-med student in Nashville and I work in the hospital (Sarah Canon: Tristar Centennial Hospital) where she was treated. Absolutely insane being able to work with the people who took these first steps!
@@artlesscalamity Ah yes, emerging technologies that alleviate pain and suffering are bad because they're only available to a select few. This is stupid, you just want to rain on people's parade. All new medications are always available to a select few at first, that's just how these things work, but in a few years they'll be more readily available and eventually they'll be inexpensive. You really need to just stop.
I have actually worked on these clinical trials in the lab, it was pretty amazing to see how fast the condition of these patients‘ blood cells improved
@@hartartiste7650 I saw them under the microscope and did a few analyses every few months over a period of 2 years. In the majority of cases they improved a whole lot in appearance and measured values compared to before treatment and didn't revert back over this two year period.
„The floodgates are open“ is the most important sentence here. This is going to rapidly produce at least better treatment for a plethora of diseases if not a cure. I would be very interested to know how the study explains certain people not seeing improvement. I don’t really understand how that’s even possible with this approach 😢
In the case of flaviogene (or whatever it is called) it might be that the editing didn't get put in the right spot, as they can't do that with this method. In the case of CRISPR, it might be that the edited cells are rejected and the faulty DNA takes over. Either way, no treatment is 100% guaranteed to work for everyone, even gene editing at this point. It will improve.
As I keep posting, because I'm so freaking happy about it, they made a drug for my extremely rare genetic disease, it uses a viral vector to convince the cells to produce the protein I am missing that causes my skin disease. The skin disease is rare, dystrophic epidermolysis bullosa, affecting 1 in 50,000 children, but it is absolutely life-changing, especially for children with the more common, and more severe variant.
@@Yvolve I use a drug that has similar mechanisms. Sometimes it works. Sometimes it doesn't. When it works it's a freaking miracle. I'm growing back a fingernail! That may not sound like much to you, but with my skin disease it's a miracle. Sometimes the drug does nothing at all.
@@edwardallenthree Congratulation! Yours is a really horrible disease and I can't imagine how you must feel for there to be an effective treatment for it. Best wishes for a bright future!
In the Netherlands a small group of patients with hereditary angioedema has been treated with this technique. The results are astonishing. It is able to give people back their lives. Of course this disease is a relatively easy fix but it is really promising.
@@aj9485the Netherlands is famously incredibly advanced with its agritech for food crops. Shockingly, given its tiny size, it's only beaten for agricultural exports by the USA - this astonishing disproportionality is due to their advanced biotech and agritech. I don't know if cannabis specifically is something that they're any better at growing than anyone else, but their laws around consumption are definitely more sensible than most
"thalassemia?" i asked myself. "why that seems to translate to 'sea blooded'. what the heck is that supposed to mean?" it's because it was first noticed among people around the Mediterranean sea. ah etymology.
There are a huge variety of Thalassaemia’s and Haemoglobinopathies all involved the 4 Globin chains of the Haemoglobin molecule. As a genetic disease it first appeared in people in the Mediterranean and then isolated in the Middle East and northern Africa. Ive worked for decades in Haematology laboratories identifying Haemoglobin Traits (mutations) and we use a specific request form with a questionnaire asking about ethic heritage and familial geography. Ancestral geography can be essential to trace the traits through familial blood lines. The ‘sea blooded” individuals are now so important that every clinical pathology practice provides testing to identify their blood.
before i became chronically ill and disabled i was studying to be a geneticist to do exactly this. I first heard of CRISPR when i was in high school and couldnt wait for it to be used to treat conditions we've had very limited ways of treating. while i havent been able to contribute to research myself im still super hyped to hear about these advancements! i look forward to hearing more about this in the future 🥰
I'm don't know what you disability is, but I think you have a great opportunity to still contribute in nonclinical ways. Like communicating new innovations in the field with future recipients. Leverage some AI tools and you might be surprised what you can accomplish in a short time. 😊
I think the same as the other answer here! I do also not know what your condition is but if you are able or have technology that is able to comment, write on a phone, tablet or whatever you are using, you can already do so much with that!! Good luck in your future with your goal!
There are so many ways you can be a part of the journey! I also heard of CRISPR in high school and decided then that I wanted to be involved in the field of genetics. I work in gene therapy now, and there are so many roles outside of the lab that are just as important. I hope more treatments become available to you and that you are doing well!
I had a bone marrow transplant 15 years ago for an autoimmune disease called aplastic anemia. Thankfully I have a lot of siblings and one of them was a perfect match so I didn’t have to wait on the donor list. It’s cool that now people are getting a chance at similar results without needing a donor because you are the donor. Great stuff 👍
I had aplastic anemia as well, and had my BMT 12 years ago. Unfortunately, neither of my brothers were a marrow match, but I was one of the lucky ones who got a donor pretty quickly. Then again, I believe I was moved to the top of the list because it was discovered I had PNH as well, which was why my two attempts at immune suppressant therapy relapsed.
Yup. Exactly this. And thats exactly why and where folks go wrong when it comes to altruistic anarchy and capital based ownership, the two are not compatable. I.E. you cannot have good results that give more freedoms and lower costs and have a capital based free-market. Capital ownership is spending money to earn money in perpetuity. I.E. Capitalism, landords, wall street. Risk or no. altruistic anarchy bends jeavily towards getting rid of ownership (as we know it) and money altogether, rewards are dolled out by reputation that can be tracked and sorted out however works best for the supplier of these luxuries; I.E. credits; wanted posters (for good, services and bringing a stop to public nuisance's), thank you letters, and contracts to supply for supplies in any or all combinations worked out between supplier and end user. And honestly after seeing so many companies go bust because they could not pay back thier investors fast enough anarchy seems more stable and innovative to me, even if it means a more inconveiant shopping experience.
Sorry, but the only thing this technique can do is trade your cancer for AIDS. There is some ongoing research that will hopefully allow you to trade it in for SaRS one day, but that's still in the early stages.
I'm hoping that one day my damaged spinal cord (C2 SCI) might be repairable or regenerable so I can move again. Maybe CRISPR holds the key, maybe something else, but I'm not holding my breath. Always good to hear when some kind of STEM advancement leaves just a few less people suffering in the world.
I have 0 experience with this and have only seen athletes do this, but it seems stem cell treatment can help heal otherwise damaged parts. I don't know about nerve damage, but it might be worth looking into if you haven't already. I hope science will find a way to fix damaged nerves and spinal cords, so many people's lives would be changed, as it would yours.
You should look at the work of Dr. David Sinclair if you haven’t already, he was able to regenerate the optic nerve after severing it in both mice and primates. I’m trying to remain hopeful as I also have nerve damage.
Crispr probably isn't going to be the one, but stem cell treatment might! I have CCI, so I live in fear of a C1/C2 SCI every day, so I've been frantically googling every time my symptoms get bad
It's wild that when he says "If I had a nickel for every gene editing treatment for sickle cell approved in December 2023... Well, You know how this one goes." The assumed conclusion is the Phineas and Ferb reference "I'd have two nickels, which isn't a lot but it's weird that it happened twice" instead of the original idiom "I'd be rich". The Phineas and Ferb joke has replaced the (maybe, probably, in it's modern incarnation) Mark Twain stock phrase and now "If I had a nickel for every time" implies by default it's happened 2 times not many
I had no idea what he was talking about - I thought he meant he'd only have one. The way I've heard it most commonly is "If I had a penny/pound for every time X has happened then I could afford Y.", usually where Y is comically low value.
@@hannahk1306 that's exactly what the top comment was talking about; the new version of the saying has started to replace the old version that you remember. In the video the joke was that its strange that there were 2 gene therapy treatments for sickle cell approved in December 2023
The updated one is always stated in the context of something extremely specific while the original is always more general so you don't even need the context. I bet we start seeing it in the decent amount of money but not huge amount too. "If I had a nickel for every time Boeing airplanes were grounded in the last 5 years, I could go get a candy bar, which is entirely too many nickels.". Or something like that.
I am a pre-med student in Nashville and I work in the hospital (Sarah Canon: Tristar Centennial Hospital) where she was treated. Absolutely insane being able to work with the people who took these first steps!
@@KyoSkyz I don't have that much say in research treatments, however as a pre-med student I plan on shadowing with the oncology department. if I learn anything I will return to this comment
I have a genetic disorder called Ehlers-Danlos Syndrome that effects the collagen of every cell, with varieties referring to which cells are the most effected. One variety of it is vascular and causes patients to die of ruptured aortas. Another variety, mine, causes loose joints, chronic pain, and repeated dislocations. I'd do (almost) anything to make the pain go away. Not sure there is anything to be done about the already damaged joints, but body-wide working fascia? Sign me up!
Wow! I have Dystrophic Epidermolysis Bullosa, which "only" affects Collagen 7a. There's a viral vector treatment now for me, it uses the herpes virus. I don't know if that would work for you. I fear when I hear descriptions of many of these disease that the viral vector option which is so easy for me would require a bone marrow transplant for you. I can only tell you, it's coming. And keep looking, I didn't sign up for the trial, and I really wish I had. It would have made the trial better, if not me.
My family have Marfan syndrome, which is a different connective tissue disorder. I'm the only one who doesn't have it so I don't need gene therapy for that myself but I'd love to *not* live in fear of my family members dropping dead from aneurysms.
The people taking part in this experiment are legends and heroes to me. I've been waiting and waiting for this to become available for myself. I've got an auto-immune disease (with no cure) called Ankylosing Spondylitis. I'm one of the lucky few where treatment is difficult to get to work and my disease is significantly more aggressive and advanced than what others have. CRISPR will theoretically fix me, but until now it's never been allowed to be used in humans. If this works for these patients, then there's hope researchers will work on a treatment for me to finally cure me. I can't wait at all. CRISPR is the only reason I haven't taken my wheelchair to the edge of a cliff and forgot the brakes. Speedy, safe and problem free recovery to everyone involved!
There's a high chance that this kind of research will be accelerated with AI. Healthcare industry will be flooded with new cures and treatments. Keep pushing forward, unlike for many patients in the past, you have the best odds.
I really, Really hope they can insert the missing Proteins in My DNA soon!! Am missing 2 out of the 4 Proteins in My DNA that makeupmyautoimmune system; I'm the only person in the world who's been found to be missing more than one. .. it doesn't even have a name yet! But im so happy that CRISPER is finally in the hands of doctors who can use it to help people!!! 🙌 😊🎉
I really hope that they develop a treatment for you too! Treatments based on viral vectors are mature. I envision over the next 5 years virtually every genetic disease will either have an ongoing trial, or a treatment. There's still a long ways to go. Getting a virus to infect the cells that are problematic isn't always easy. As you can see in this case, both treatments, require a bone marrow transplant to work. That isn't tenable for most humans. The treatment for my skin disease, a viral vector treatment, the first topical ever approved, works only on open wounds. I didn't get treatment this week, because all of my wounds are slightly healed and it wouldn't do any good. They're hoping to be able to develop more, because one of the conditions that they really want to treat is wrinkles. The problem is getting the skin infected, when the epidermis is extremely effective at stopping the herpes virus.
@@edwardallenthreeThis is exactly what I want my Ph.D in. T-VEC is an incredible advancement but definitely has its drawbacks. We only in the past few years have been able to get past the biggest bottleneck to these therapies (getting accurate protein structures quickly) and my assumption similar to yours in that the next decade or so will have these therapies cure nearly all genetic diseases and then some. My current lab is even hopeful that the current gene we are working with can eventually work similarly to a broad spectrum antiviral.
Ive beem on gene therapy since 2016 for cystic Fibrosis. Vertex started small, saw success with smaller generic defects then dove in for delta f508...it worked too. Its a game changer to say the least
I've never been so happy about some science news, this will grow exponentially, and one day soon this is the norm, we're about to enter a whole new world and I couldn't wish for something more exciting to witness
Did you hear the one about the deaf kid, who hearx his first sound at 12 years old, not with the help of an implant but with a viral vector genetic modification? What about the people with the generative eye disease who are in trials and regaining their sight? I am growing a fingernail back, mild in comparison, but a significant achievement with my severe genetic skin disorder. We need to develop these treatments for as many diseases as we can as quickly as we can at the same time. The order we do them needs to be the order in which we can effectively treat them. If it's easy, we should do it. If it's hard, we should try and work until it is not. We need to take advantage of synergies when we can. The dirty secret behind the treatment for my skin disease is that the company behind it hopes to leverage the technology to treat another genetic skin condition, wrinkles. This energy provides funding, motivation, and a financial reward for success. Any place that synergies like this can be found should be leveraged. Should do whatever we can to help people with rare diseases, and gosh darn it, I've never been so optimistic! Did I mention I grew a fingernail back? Thank you Vyjuvek. Thank you genetic treatment.
it’d be really cool if there’s a way to use gene therapy for endometriosis or multiple sclerosis i have both and everything besides symptom control and surgery looks kinda bleak at the moment
@cwcorella.""AI"" aren't a thing yet. There's just bad algorithms being given too much credit, power, and attention. You are right that its too slow, though.
@cwcorella.If you knew how incredibly complex Human DNA is and the immense risks of editing it, you wouldn't be saying that we are slow with this technology. Mistakes could have disastrous consequences for the patients
I’m there with you. A complete hysterectomy helped with the endometriosis except for what’s on my intestines that they couldn’t remove all of. It made a huge difference for the better for me. I have MS too. I’m now allergic to direct sunlight and a ton of foods! It’s been a long battle. I’m tired and ready for a cure!
Really hope to see some treatments for Elhers-Danlos syndrome, absolutely debilitating condition with multiple types and specific complications associated with them
Its weird listening to you talk about things related to stuff ive worked on and with in the past. The biggest concern I found with these types of gene editing was definitely the on/off target editing going on in the cells. Even though the process is precise, there is always the chance of it not going to the right target sequence such as cutting too early or cutting or late or even in the whole wrong area and causing unexpected mutations to protein encoding and such. Getting the results back the biggest statisic I would want to see is the ratio of On/Unaltered/Off targeted that occured. It seems their method must have been reliable enough due to most patients feeling well after treatment but I still worry for them due to the as stated blood cancer potential. Thanks for making this video!
@@b.a.erlebacher1139but because it’s done outside the body, they can run tests to make sure that only the target gene has been altered, and make any necessary changes if there are any errors, so there shouldn’t be errors being introduced if they do it right.
@@Rwdphotos that's probably why it takes 6 months to get the edited stem cells back in the patient. To grow enough of the corrected cells and minimal sick/off targeted cells. You should know where most potential off targeted sites occur but I don't know if there is a 100 percent certainty of it not cutting somewhere else in the sequence
Well researched, executed, and cited presentation. This is a fantastic video and I think the best of what this channel has to offer. Keep up the good work!
Love this guy! Making science fun, not that it ever wasn't, again. Reid is very easy on the eyes and always something interesting to talk about. Keep up the great work. Patron here I come ...
I was wishing for something like this around 40 years ago when my son was diagnosed with "Bubble Boy disease" and we wouldn't have had to go through the Bone Marrow Transplant at Texas Children's.
@@maggierobertson2962 His body rejected the Transplant but it caused his system to start working a little. If they would do a second Transplant they would have to give him chemo and completely wipe out the immunity he had and we didn't want to do that. He lived until he was 18 but he had to get IV gamma globulin every 3 weeks the rest of his life. He didn't really have a "normal" life which is what he really wanted.
Wow, this is a great episode! I especially like how you described the treatment and mentioned the cost! I had no idea how gene editing was done on an existing person, but I learned something yay! Also yay Reid 😊
Thanks for making this video. The most interesting part for me was learning how the actual gene editing is done outside the body. It helps me to understand that there are fewer risks than turning some nightmare gene modifier loose in a patient's body.
Incredible news! I'm amazed at how low risk it is from a layman's perspective. As far as the issue is with the benefits not carrying over to eggs and sperm, widely accessible prenatal DNA testing like in Iceland could help parents ensure their baby doesn't have Sickle Cell or other genetic disorders.
The therapy not affecting eggs and sperm is a feature, not a bug in this instance. Modification of gametes (egg and sperm) is hotly debated ethical issue. The general consensus is that the risk of off target modification (like where he mentioned DNA incorporating anywhere in the genome and potentially breaking something) is regarded as still too high with crispr. Essentially, the risk to a potential person who cannot consent to that risk is just too high for gamete modification. Beyond that, the fears that such a modification will open up the door to designer babies is something that is discussed frequently. While it is extremely rare to find people who would support this, changing regulation of gene therapies to allow changes that can be passed to offspring will, as he said, open the floodgates, and not all of the people who take advantage of that will be looking to just change genes to cure future babies. Your point about preimplantation/prenatal genetic diagnosis(or preimplantation genetic testing) (PGD or PGT) is hugely important. There are still ethical concerns that genetic counselors have to account for, such as people wanting to implant only children of a certain sex or with certain desirable traits, but the potential for PGD to massively reduce the risk of passing on genetic disorders to ones children can have huge benefits. This is especially true as, for many disorders, parents are not guaranteed to pass on a disease to their children. Again, there is a lot of sensitive discussions that have to be had about what traits are okay to select for against, and how do we as a society treat these genetic disorders in such a way that we do not devalue the people who have already been born who have those traits. I am still hopeful such technology can help us achieve a healthier happier future for our children.
Well I’m sure there’s still a decent amount of risk, considering the chemo therapy would wreck havoc on the immune system for a while, as well as impact organs if not enough new blood cells are able to be made by the body. They would likely need to be infused with blood during the entire process and be immunocompromised, so any infection would be an incredibly serious event.
@@brianchew2565Thank you for breaking down such a broad set of issues into only 2 paragraphs - I read it all. The ethical issues are definitely something I was aware of, but you elaborated on some finer points that I needed to hear. I think it's paramount that if society does go down the path of widescale prenatal testing, we make it clear that people still living with disorders - and the qualities of their lives - are still valued.
@@brianchew2565I hear the slippery slope argument, but I can't help but see it as an expensive and physically intensive cure that each generation would have to undergo separately
@@brianchew2565 Good summary! For me, the biggest ethical issue is access. These technologies are pro-human only to the extent that all humans can access them.
Now do an episode about how MDMA/psilocybin are being approved by the FDA for all kinds of treatments. In Australia they’re already legalized for certain treatments of PTSD/end of life depression due to cancer.
Seconding this!! I happen to know someone who was involved in one of the most recent studies about it and it's unbelievably exciting news. It could be such a massive breakthrough in PTSD treatment
i heard about psilocybin being effective for cluster headaches and migraine disorders, i get brain stem migraines with aura i think hemiplegic migraine facial migraines(in the area around your eyes and nose it feels like it’s coming from your sinuses) vestibular migraine and neurological issues from multiple sclerosis including cluster headaches and trigeminal and occipital neuralgia and conventional treatment like nsaids don’t really work well for me so i’m genuinely curious
more like unbelievable how slow it is, the first genome editing techniques were invented in the 80s, bioethicists and other clueless groups of bureaucratic morons have been holding the field back
Incredible. This is the beginning of something incredible beyond what we can even begin to imagine. I’m so excited to see this technology progress and continue to save and change lives for the better. Yay science!
The viral vector treatment is freaking amazing. It's already been approved for my disease, dystrophic epidermolysis bullosa, and there are trials for genetic disorders causing blindness, deafness and many many other severe disabilities. Treatments for all of these are just around the corner.
Hi, it’s a shame that it’s likely to be so expensive in the U.S. and hence likely to be unfeasible for the majority. Similarly here in the UK NICE decides if the NHS will be able to offer expensive treatments. I’m not sure what the answer to this is but hearing of exciting new treatments for some awful diseases is always tempered by the reality of cost smacking me in the face. Hopefully I am wrong and it is affordable for everyone.
My insurance company was more excited than I was to cover my $25k a week genetic treatment. I have theories as to why, but, what it comes down to, is in both the UK and the United States it is not the rare diseases which are causing our health systems to be financially insolvent.
In this case, its a result of the actual cost of development. Developing a therapeutic like this already has massive costs to a company, that they would like to recoup from patients so they further develop new therapies, but the technology to carry out this therapy is also very expensive, and the cost to maintain the standards for a lab to do this and ensure the therapy is safe for a patient is also hugely expensive. At the moment, that means that most gene therapies or stem cell therapies will be wildly expensive. This leads to a lot of discussion among researchers about what therapies are best to pursue, as we want to help as many people impacted by diseases or disorders as we can, but further development of this technology also has a high likelihood of decreasing the cost of future therapies. All of this is to say that is sucks that this is so expensive at the moment, but there is great hope that costs will decrease in the future, and scientists who develop these (at least within public sector research) are actively looking at ways to make these more widely available all the time.
Searching for affordable alternatives is a big part of science research in general. It's expensive right now, but going by past experience, that will definitely change in the future. E.g. the cost of sequencing a genome nowadays is a drop in the bucket compared to how mind-numbingly expensive it used to be at the advent of that technology
Great video! I love that you went into details of how clinical trials work and pros and cons as well as the basic information about how these techniques work. I can't wait to show this video to my intro bio students! 1 tiny mistake I noticed: Early in the video, the 2 alleles for the hemoglobin gene were shown on sister chromatids of 1 chromosome, instead of on homologous chromosomes.
Chemo/stem cell transplant part isnt as bad as it may sound for most, I didnt have any adjustments to my stem cells but I went through the filter/chemo process to treat lymphoma cancer. Not going overly into detail to keep it shorter, the collection of stem cells takes anywhere from one to several 5 hour hospital visits, for the vast majority its 1 or two days and you go home immediately after they are done. The chemotherapy requires a significantly longer hospital visit, but thats because you lose your immune system and by staying at the hospital you can isolate yourself. Assuming you dont catch anything the other main side effect is fatigue, you will have very little energy immediately after and some fatigue/weaker immune system issues for several months afterwards, but not so bad you cant be at home.
All these major breakthroughs mean is that it will be heavily monetized and then offered to only the smallest subset of people who have immense wealth and resources
I was in girl scouts with a girl who had sickle cell disease (at the time i knew it as sickle cell anemia). I recall her going through a BMT treatment. I haven't seen her in probably 20 years but i hope she's doing well.
Thanks for this, its extremely rare to hear firsthand from someone who is directly benefitting from our work, when working in a lab/cleanroom i find we become disjointed with the end goal and this means a lot!
Sickle cell is prevalent in areas with high Malaria burdens since Sickle cell disease confers some resistance to the Plasmodium parasites that cause Malaria. I’d be curious to know if this treatment can remove the negative effects of the disease without removing the resistance to Malaria. If the treatment does cause a reduction in Malaria resistance, it could cause more harm than good if deployed widely in Malaria endemic areas without additional Malaria mitigation work.
@@CaTastrophy427 I imagine most of the people who can afford this don’t live in places where it matters. It will matter if mass deployment ever becomes possible, which seems probable over time.
It's important to remember that tech of ANY kind is ridiculously expensive at first, but as the tech gets refined, the price comes down. These treatments are in the millions currently, but I expect in 5-10 years it will be in range of us commoners.
I would love to see you cover how Vyjuvek from Krystal Biotech works for EB. It is a virus vector treatment, the first topical ever approved. I have DDEB and it "helps," but it is life changing for RDEB.
I desperately hope the cost changes soon. A cure for chronic disease like this is miraculous... but if that cure can be withheld so easily, the shadow of eugenics always looms. I'm sure there's going to be significant research about making CRISPR treatments more cost effective soon, so here's hoping.
Family member had a blood disorder and had to get a bone marrow transplant. This seems effectively the same process but instead of a donor donating stem cells, the patient's own stem cells are used. This is good though for not having to worry about rejection. GVHD & chemo were the worse parts. This removes one from the equation.
They haven't cured mine, but the viral vector treatment was developed and it does work as a treatment for my genetic disease (Dystrophic Epidermolysis Bullosa). I hope they cure yours.
Many years ago we had a young African-American male come into the hospital in respiratory crisis form a Colorado mountain town. Ironically (as you will see in a minute) he was flown in Flight for Life. It was determined that he was in sickle cell crisis secondary to altitude. The recovery hospital was at an altitude of over 6000 feet and he was stabilized there. Once he was well enough to leave he was told he could NOT go get his car and that he needed to take a VERY long way home avoiding all mountain passes. I've often wondered how he was doing.
With regards to sickle cell. It appears to be concentrated in Certain parts of Sub-Saharan Africa. With Malaria being an issue, this would make sense. either the Malaria gets you, or the Anemia. I am wondering if there are different health outcomes between those who live a more indigenous lifestyle (local foods/medicines/herbs/work habits/sleep habits) who have sickle-cell compared to those afflicted who live in Urban environments.
The fact that Heterozygotes of the sickle cell disease persist to this day show that it was slightly selected for in terms of population genetics. Those who could resist malaria survived and were able to pass on their genes. This explains why we see it more in these populations, but having both copies of the gene is still detrimental no matter the lifestyle. Without access to treatment, I'm sure having sickle cell is life threatening, it's just a matter of time.
@@billybob7688453 Im curious about the local herbs, remedies used. Ive been reading up on different non-western medicines. this is just another one to add to the list :)
All great things start with humble beginnings, Crisper might only be helping a handful of people right now but soon it will be helping the whole world.
My conclusion is that this CRISPR-CAS9 treatment shares a lot of similarities with bone marrow transplantation of a matching donor. However, the donor is the patient itself this way. There are a few creases to iron out, but I believe this has a great future.
I have a blood disorder (von Willebrands disease) so this is great news. I can imagine the utility of CRISPR will expand pretty rapidly now that it’s already being used clinically.
Thinking about it.. I *did* hear about Crispr 10 years ago. I always wondered why they didn't deploy it sooner! I know testing is important but this can change so much for the better!! :D
As someone who had 2 different bone marrow transplants (for myeloma [plasma cell cancer], but same process), it's always weird to see what else that process can be used to treat. The process is pretty rough, and when a donor is involved the long lasting side effects (GVHD) can be rough too, but when the alternative is worse, it's still worth it. Hopefully we come up with a safe way to do the gene editing inside the body instead of having to do the whole transplant thing.
Well it's progress, but it sounds like it is still an extremely arduous treatment. A significant advancement, but probably doesn't open the floodgates to use CRISPR for all sorts of problems.
The price tag is for all of us, not you. That is why we have society. I will gladly pay more in insurance or taxes to cover you, and I hope you would extend me the same courtesy. I was shocked when my insurance covered a genetic treatment that is similarly expensive for me (25k/week ongoing treatment). I felt guilty. One of my docs said, "don't feel guilty about how much insurance pays on your behalf." Another said, "you are an extraordinary person with an extraordinary disease that requires extraordinary treatment, don't feel bad about people helping you." That having been said, if I can skip a treatment, I do.
Baby blood instructions located different than where adult blood instructions are. Is that so the baby has a more likely chance to match the mom's blood type and antigens on her blood?
1:58 The story of the adaptation/mutation of hemoglobin beta to protect against the billions of historic deaths from malaria should not be wholly divorced from the story of sickle cell disease and its treatment: the gene is a trade-off, not a defect.
Very exciting. Also very sad knowing there is a cure for something, but few can afford. Hopefully in the years to come it will become more affordable so the people affected by this disease are able to get their lives back.
I'm going to venture to say that any American who wants to pursue this treatment will almost certainly find whatever funding they need, and I say that from experience of having a rare disease and needing funding to cover a new genetic drug treatment. The problem with these treatments at this time is not their expense. It's that, right now, so few people actually qualify to get it.
Wow, this prospective technology sent me down the route of becoming a scientist a decade ago - and now, working in clinical immunohematology, it’s more exciting than I can put into words knowing that some of my sickle patients may soon be able to stop coming in for their exchange transfusions!!
For starters we have other sophisticated new treatments that needs less intrusion than this but since these treatments well studied and got enough trials they can get pass. But once floodgates open we can see more sophisticated cell and tissue targeted gene therapies without chemos or lengty processes in the field. In medicine tech always go way ahead than the aproved trwatments due ro lengthy process of getting it aproved
I really wish I could get involved in a company that is doing this kind of research, but I just have a background in data science and nothing in biology.
I've been researching everything we know about crispr since i was eleven, i thought it may be able to help with my mom's rheumatoid arthritis (it might be possible), but now more than ever since I've learned i have multiple genetic chronic conditions including Hypermobile Ehlers Danlos syndrome, MCAS, and a few others, i am excited to see how it progresses
I love that f*cking shirt. Where can I get one? Oh, and yes, CRISPR is an awesome look into our future. Keep pumping out these great videos. Love yous at SciShow. Seriously about the shirt bro.
12:11 In this case, there is no way around it being so expensive. It is the cutting edge of medical technology and that is going to cost a lot of money. It has to start somewhere and some people are going to pay through the nose to have this treatment, which will make it cheaper over time. There will be a machine that can take over the laborious manual tasks and cut a lot of cost, and probably time. Treatment being expensive is the norm in medical science, as is the price coming down.
I mean, yes, BUT the question of who foots the bill is incredibly relevant. Some countries force individuals to pay a la carte for treatments, other countries socialize all/most healthcare costs. So, expensive doesn't have to mean unaffordable by default. The outcome for individuals is very sensitive to the economics of healthcare in a given country.
There is a common mistake in this video. Both chromatids of the chromosome sketched at 1:53 have the same identical copies of any given gene. If there is a different copy it is located on the other chromosome of the pair.
I have one copy of the sickle cell gene. When my mom married my dad, they had tests done on him to ensure there was no chance he'd have another copy of the gene, as to avoid children having it. It's nice to know the next generations of my family will not have to care that much about this anymore
I am a lab tech that makes the Casgevy product. It's extremely labour intensive and not really open to automation, so I'm afraid the price isn't going to go down any time soon
Ok this is pretty off topic but I saw the bell-ringing stock footage and finally realized what that bell for is at my cancer center lol. I go there a lot because it's technically a cancer and hemotology clinic and I have to get fairly regular iron infusions for my anemia.
CRISPR & AI could advance medicine faster than ever before, as information now doubles every 3 yrs its incredible to imagine finding the cures that will end suffering for humanity. Imagine lowering the cost of cures are the norm rather than expensive pharmaceutical treatments. The benefits definitely outweigh the risk.
I'm glad they didn't shy away from the cost of this treatment. It is eyewatering. And it's even more concerning that there isn't much to be said about how it could be reduced over time. And there's the other question of how cheap you can go without jeopardizing treatment integrity.
We need to put in at least 1-3Mill a year into this because this is the beginning to ending cancer and other problems in the body. This might also go hand in hand with curing aging and gene editing so that we can stay younger much longer. I hope we get a lot more news on this as the years go on and maybe get a yearly update on it’s progress.
Maybe one day it could help people with my condition, too. It’s exciting! I hope everyone who is having this treatment live long, happy, fulfilling lives with no complications from their original disease or the treatment!
A few months ago, my medical school invited the first person treated for sickle cell disease with this technology to lecture about their experience, and I had the opportunity to meet them. Indeed, we live in exciting times!
Taeqwanmouse2105
I am a pre-med student in Nashville and I work in the hospital (Sarah Canon: Tristar Centennial Hospital) where she was treated. Absolutely insane being able to work with the people who took these first steps!
Wow!!
Except for the $2.2 million price tag…
The rich live in exciting times. The rest of us live under a cruel, for-profit healthcare system.
@@artlesscalamity Ah yes, emerging technologies that alleviate pain and suffering are bad because they're only available to a select few.
This is stupid, you just want to rain on people's parade. All new medications are always available to a select few at first, that's just how these things work, but in a few years they'll be more readily available and eventually they'll be inexpensive. You really need to just stop.
I have actually worked on these clinical trials in the lab, it was pretty amazing to see how fast the condition of these patients‘ blood cells improved
Wow! How exciting to be able to work on this!!!
Temporarily, cosmetically or long term?
@@hartartiste7650 I saw them under the microscope and did a few analyses every few months over a period of 2 years. In the majority of cases they improved a whole lot in appearance and measured values compared to before treatment and didn't revert back over this two year period.
@@hartartiste7650 I have tried to answer this question but for whatever reason my comments keep getting deleted. 🙄
So, tell us, when can we all become undying gods?
„The floodgates are open“ is the most important sentence here. This is going to rapidly produce at least better treatment for a plethora of diseases if not a cure.
I would be very interested to know how the study explains certain people not seeing improvement. I don’t really understand how that’s even possible with this approach 😢
In the case of flaviogene (or whatever it is called) it might be that the editing didn't get put in the right spot, as they can't do that with this method.
In the case of CRISPR, it might be that the edited cells are rejected and the faulty DNA takes over.
Either way, no treatment is 100% guaranteed to work for everyone, even gene editing at this point. It will improve.
As I keep posting, because I'm so freaking happy about it, they made a drug for my extremely rare genetic disease, it uses a viral vector to convince the cells to produce the protein I am missing that causes my skin disease. The skin disease is rare, dystrophic epidermolysis bullosa, affecting 1 in 50,000 children, but it is absolutely life-changing, especially for children with the more common, and more severe variant.
@@Yvolve I use a drug that has similar mechanisms. Sometimes it works. Sometimes it doesn't. When it works it's a freaking miracle. I'm growing back a fingernail! That may not sound like much to you, but with my skin disease it's a miracle. Sometimes the drug does nothing at all.
@@edwardallenthree Congratulation! Yours is a really horrible disease and I can't imagine how you must feel for there to be an effective treatment for it. Best wishes for a bright future!
@@Yvolve Sounds like a lot of stuff can go really wrong here. Imagine the things that´s gonna wash up on Plumb Island when the CIA eyes this tech. lol
In the Netherlands a small group of patients with hereditary angioedema has been treated with this technique. The results are astonishing. It is able to give people back their lives. Of course this disease is a relatively easy fix but it is really promising.
😂
So proud to be part of the biotech force of NL! We are going strong, also with crops 💪
@@nigtendos what kind of crops 🤣
@@aj9485stroopwafels.
@@aj9485the Netherlands is famously incredibly advanced with its agritech for food crops. Shockingly, given its tiny size, it's only beaten for agricultural exports by the USA - this astonishing disproportionality is due to their advanced biotech and agritech.
I don't know if cannabis specifically is something that they're any better at growing than anyone else, but their laws around consumption are definitely more sensible than most
"thalassemia?" i asked myself. "why that seems to translate to 'sea blooded'. what the heck is that supposed to mean?" it's because it was first noticed among people around the Mediterranean sea. ah etymology.
thalass meaning sea, emia meaning presence in blood
Maybe also useful to describe people full of salt
@@Appletank8You would have a lot of salt if you had part of the mediterranean sea in your blood
There are a huge variety of Thalassaemia’s and Haemoglobinopathies all involved the 4 Globin chains of the Haemoglobin molecule. As a genetic disease it first appeared in people in the Mediterranean and then isolated in the Middle East and northern Africa. Ive worked for decades in Haematology laboratories identifying Haemoglobin Traits (mutations) and we use a specific request form with a questionnaire asking about ethic heritage and familial geography. Ancestral geography can be essential to trace the traits through familial blood lines. The ‘sea blooded” individuals are now so important that every clinical pathology practice provides testing to identify their blood.
@@Envy_May Damn. You beat me to it he he
before i became chronically ill and disabled i was studying to be a geneticist to do exactly this. I first heard of CRISPR when i was in high school and couldnt wait for it to be used to treat conditions we've had very limited ways of treating. while i havent been able to contribute to research myself im still super hyped to hear about these advancements!
i look forward to hearing more about this in the future 🥰
I'm don't know what you disability is, but I think you have a great opportunity to still contribute in nonclinical ways. Like communicating new innovations in the field with future recipients. Leverage some AI tools and you might be surprised what you can accomplish in a short time. 😊
I think the same as the other answer here! I do also not know what your condition is but if you are able or have technology that is able to comment, write on a phone, tablet or whatever you are using, you can already do so much with that!! Good luck in your future with your goal!
There are so many ways you can be a part of the journey! I also heard of CRISPR in high school and decided then that I wanted to be involved in the field of genetics. I work in gene therapy now, and there are so many roles outside of the lab that are just as important. I hope more treatments become available to you and that you are doing well!
@@NenJiDaPassiv they could have the opportunity to contribute in clinical ways too
Oh I'm talking to kids nvm. Yeah I was well into my 20s when Crispr was discovered and developed.
I had a bone marrow transplant 15 years ago for an autoimmune disease called aplastic anemia. Thankfully I have a lot of siblings and one of them was a perfect match so I didn’t have to wait on the donor list. It’s cool that now people are getting a chance at similar results without needing a donor because you are the donor. Great stuff 👍
I had aplastic anemia as well, and had my BMT 12 years ago. Unfortunately, neither of my brothers were a marrow match, but I was one of the lucky ones who got a donor pretty quickly. Then again, I believe I was moved to the top of the list because it was discovered I had PNH as well, which was why my two attempts at immune suppressant therapy relapsed.
To be fair, these things tend to get cheaper as time goes on.*
*So long as the development process is not held by a singular company.
Hopefully the two options compete each other into affordability.
Yup. Exactly this.
And thats exactly why and where folks go wrong when it comes to altruistic anarchy and capital based ownership, the two are not compatable.
I.E. you cannot have good results that give more freedoms and lower costs and have a capital based free-market.
Capital ownership is spending money to earn money in perpetuity. I.E. Capitalism, landords, wall street. Risk or no.
altruistic anarchy bends jeavily towards getting rid of ownership (as we know it) and money altogether, rewards are dolled out by reputation that can be tracked and sorted out however works best for the supplier of these luxuries; I.E. credits; wanted posters (for good, services and bringing a stop to public nuisance's), thank you letters, and contracts to supply for supplies in any or all combinations worked out between supplier and end user.
And honestly after seeing so many companies go bust because they could not pay back thier investors fast enough anarchy seems more stable and innovative to me, even if it means a more inconveiant shopping experience.
But think of the profits for that monopoly. Surely more profits for the already wealthy is a worth literally holding back global human progress
I think institutions that have the ability to heal people should do so for free. You know, innate right to longevity and all that.
Luckily, as CRISPR is not controlled by one company, that shouldn’t be an issue!
Hope this will cure my leukaemia soon
Hang in there, best wishes.
You have my prayers, stranger. Be strong.
Have you tried Rhino Horn?
Jk
It would be nice for y'all but you guys know it will cost a fortune and insurance won't pay because "it's a new experimental procedure"
Sorry, but the only thing this technique can do is trade your cancer for AIDS. There is some ongoing research that will hopefully allow you to trade it in for SaRS one day, but that's still in the early stages.
Seeing a SciShow episode on CRISPR is the reason is wanted to study genetics. And now nearly done with my degree. 😊
I'm hoping that one day my damaged spinal cord (C2 SCI) might be repairable or regenerable so I can move again. Maybe CRISPR holds the key, maybe something else, but I'm not holding my breath. Always good to hear when some kind of STEM advancement leaves just a few less people suffering in the world.
I have 0 experience with this and have only seen athletes do this, but it seems stem cell treatment can help heal otherwise damaged parts. I don't know about nerve damage, but it might be worth looking into if you haven't already.
I hope science will find a way to fix damaged nerves and spinal cords, so many people's lives would be changed, as it would yours.
Well I hope it does man, science is advancing quickly and it is honestly very exciting
Perhaps neurolink one day would be more viable for a spinal injury.
You should look at the work of Dr. David Sinclair if you haven’t already, he was able to regenerate the optic nerve after severing it in both mice and primates. I’m trying to remain hopeful as I also have nerve damage.
Crispr probably isn't going to be the one, but stem cell treatment might!
I have CCI, so I live in fear of a C1/C2 SCI every day, so I've been frantically googling every time my symptoms get bad
It's wild that when he says "If I had a nickel for every gene editing treatment for sickle cell approved in December 2023... Well, You know how this one goes." The assumed conclusion is the Phineas and Ferb reference "I'd have two nickels, which isn't a lot but it's weird that it happened twice" instead of the original idiom "I'd be rich". The Phineas and Ferb joke has replaced the (maybe, probably, in it's modern incarnation) Mark Twain stock phrase and now "If I had a nickel for every time" implies by default it's happened 2 times not many
to be fair the P&F one is a lot funnier and more commonly applicable
I had no idea what he was talking about - I thought he meant he'd only have one.
The way I've heard it most commonly is "If I had a penny/pound for every time X has happened then I could afford Y.", usually where Y is comically low value.
@@hannahk1306 that's exactly what the top comment was talking about; the new version of the saying has started to replace the old version that you remember. In the video the joke was that its strange that there were 2 gene therapy treatments for sickle cell approved in December 2023
The updated one is always stated in the context of something extremely specific while the original is always more general so you don't even need the context. I bet we start seeing it in the decent amount of money but not huge amount too. "If I had a nickel for every time Boeing airplanes were grounded in the last 5 years, I could go get a candy bar, which is entirely too many nickels.". Or something like that.
I didnt realise the PnF joke was commonly quoted.
As someone living with terminal ALS this gives me hope that CRISPR can evolve enough to cure this horrible illness too.
I am a pre-med student in Nashville and I work in the hospital (Sarah Canon: Tristar Centennial Hospital) where she was treated. Absolutely insane being able to work with the people who took these first steps!
Marvelous ❤
Clever YT name for your profession. Very clever!
I need to to get this treatment I have sickle cell trying to get in contact with someone that could help me get crispr therapy
@@KyoSkyz I don't have that much say in research treatments, however as a pre-med student I plan on shadowing with the oncology department. if I learn anything I will return to this comment
@@MicrowaveOvenVideo thank you
I have a genetic disorder called Ehlers-Danlos Syndrome that effects the collagen of every cell, with varieties referring to which cells are the most effected. One variety of it is vascular and causes patients to die of ruptured aortas. Another variety, mine, causes loose joints, chronic pain, and repeated dislocations. I'd do (almost) anything to make the pain go away. Not sure there is anything to be done about the already damaged joints, but body-wide working fascia? Sign me up!
Wow! I have Dystrophic Epidermolysis Bullosa, which "only" affects Collagen 7a. There's a viral vector treatment now for me, it uses the herpes virus. I don't know if that would work for you. I fear when I hear descriptions of many of these disease that the viral vector option which is so easy for me would require a bone marrow transplant for you.
I can only tell you, it's coming. And keep looking, I didn't sign up for the trial, and I really wish I had. It would have made the trial better, if not me.
I also have EDS. I'd give anything for my daughter and granddaughter to have access to a cure.
i do too, i’m not sure about it but an effective treatment besides being expected to tough it out and take analgesics all the time would be great
My family have Marfan syndrome, which is a different connective tissue disorder. I'm the only one who doesn't have it so I don't need gene therapy for that myself but I'd love to *not* live in fear of my family members dropping dead from aneurysms.
Same!
This is what technology should be used for. This is amazing.
The people taking part in this experiment are legends and heroes to me. I've been waiting and waiting for this to become available for myself.
I've got an auto-immune disease (with no cure) called Ankylosing Spondylitis. I'm one of the lucky few where treatment is difficult to get to work and my disease is significantly more aggressive and advanced than what others have.
CRISPR will theoretically fix me, but until now it's never been allowed to be used in humans. If this works for these patients, then there's hope researchers will work on a treatment for me to finally cure me.
I can't wait at all. CRISPR is the only reason I haven't taken my wheelchair to the edge of a cliff and forgot the brakes.
Speedy, safe and problem free recovery to everyone involved!
I pray for your success and recovery and wish you the best.
Im praying for it to happen! All the best to you
There's a high chance that this kind of research will be accelerated with AI.
Healthcare industry will be flooded with new cures and treatments.
Keep pushing forward, unlike for many patients in the past, you have the best odds.
My grandma has this! She is in constant misery, sending all of the luck possible to you and her
My sister may have this as well we just found out, I'm really hoping for y'alls sake that CRISPR can work it's magic.
Maybe we should thank Jennifer Doudna for inventing CRISPR, she got the Nobel Prize in chemestry and the French lady as the co-inventor.
and feng zhang for proving its use in mammalian cells
She got a French lady for inventing CRISPR?
She has dna in her name, she was destined to invent it.
Feng Zhang discovers and develops CRISPR tools with the potential to diagnose and treat disease, including disorders affecting the nervous system
@@ELYESSS
well, that makes sense now
I really, Really hope they can insert the missing Proteins in My DNA soon!! Am missing 2 out of the 4 Proteins in My DNA that makeupmyautoimmune system; I'm the only person in the world who's been found to be missing more than one. .. it doesn't even have a name yet!
But im so happy that CRISPER is finally in the hands of doctors who can use it to help people!!! 🙌 😊🎉
I really hope that they develop a treatment for you too! Treatments based on viral vectors are mature. I envision over the next 5 years virtually every genetic disease will either have an ongoing trial, or a treatment.
There's still a long ways to go. Getting a virus to infect the cells that are problematic isn't always easy. As you can see in this case, both treatments, require a bone marrow transplant to work. That isn't tenable for most humans.
The treatment for my skin disease, a viral vector treatment, the first topical ever approved, works only on open wounds. I didn't get treatment this week, because all of my wounds are slightly healed and it wouldn't do any good. They're hoping to be able to develop more, because one of the conditions that they really want to treat is wrinkles. The problem is getting the skin infected, when the epidermis is extremely effective at stopping the herpes virus.
Hope they get you a new gene soon then, also they should let you name it then! Tell them! If you’re the only one it’s officially yours!
@@edwardallenthreeThis is exactly what I want my Ph.D in. T-VEC is an incredible advancement but definitely has its drawbacks. We only in the past few years have been able to get past the biggest bottleneck to these therapies (getting accurate protein structures quickly) and my assumption similar to yours in that the next decade or so will have these therapies cure nearly all genetic diseases and then some. My current lab is even hopeful that the current gene we are working with can eventually work similarly to a broad spectrum antiviral.
I was going to say that I’d never heard of this. I hope they can find a cure for you.
It has a name, since you are the first, they will name it after you.
Ive beem on gene therapy since 2016 for cystic Fibrosis. Vertex started small, saw success with smaller generic defects then dove in for delta f508...it worked too. Its a game changer to say the least
What's your experience been with the therapy? I'd love to hear more
How has it been a game changer? Do they call it a cure? I'll have to do some research. It's a race against time for those we love.
I love that, I'm a respiratory therapist and an avid fan of gene therapy. I think in a decade CF is gonna be toast! Good luck 🤞
Crispr feels like my kid growing up... good job buddy!
I've never been so happy about some science news, this will grow exponentially, and one day soon this is the norm, we're about to enter a whole new world and I couldn't wish for something more exciting to witness
After this helps people with deadly blood disorders, I hope it can be extended to people with autoimmune disorders.
Did you hear the one about the deaf kid, who hearx his first sound at 12 years old, not with the help of an implant but with a viral vector genetic modification? What about the people with the generative eye disease who are in trials and regaining their sight? I am growing a fingernail back, mild in comparison, but a significant achievement with my severe genetic skin disorder.
We need to develop these treatments for as many diseases as we can as quickly as we can at the same time. The order we do them needs to be the order in which we can effectively treat them. If it's easy, we should do it. If it's hard, we should try and work until it is not.
We need to take advantage of synergies when we can. The dirty secret behind the treatment for my skin disease is that the company behind it hopes to leverage the technology to treat another genetic skin condition, wrinkles. This energy provides funding, motivation, and a financial reward for success. Any place that synergies like this can be found should be leveraged.
Should do whatever we can to help people with rare diseases, and gosh darn it, I've never been so optimistic!
Did I mention I grew a fingernail back? Thank you Vyjuvek. Thank you genetic treatment.
it’d be really cool if there’s a way to use gene therapy for endometriosis or multiple sclerosis i have both and everything besides symptom control and surgery looks kinda bleak at the moment
@cwcorella.""AI"" aren't a thing yet. There's just bad algorithms being given too much credit, power, and attention. You are right that its too slow, though.
@cwcorella.If you knew how incredibly complex Human DNA is and the immense risks of editing it, you wouldn't be saying that we are slow with this technology. Mistakes could have disastrous consequences for the patients
I’m there with you. A complete hysterectomy helped with the endometriosis except for what’s on my intestines that they couldn’t remove all of. It made a huge difference for the better for me. I have MS too. I’m now allergic to direct sunlight and a ton of foods! It’s been a long battle. I’m tired and ready for a cure!
Really hope to see some treatments for Elhers-Danlos syndrome, absolutely debilitating condition with multiple types and specific complications associated with them
As someone with EDS, yeah...
Hear hear! We are suffering out here...
Its weird listening to you talk about things related to stuff ive worked on and with in the past. The biggest concern I found with these types of gene editing was definitely the on/off target editing going on in the cells. Even though the process is precise, there is always the chance of it not going to the right target sequence such as cutting too early or cutting or late or even in the whole wrong area and causing unexpected mutations to protein encoding and such. Getting the results back the biggest statisic I would want to see is the ratio of On/Unaltered/Off targeted that occured. It seems their method must have been reliable enough due to most patients feeling well after treatment but I still worry for them due to the as stated blood cancer potential. Thanks for making this video!
If the blood is treated outside of the body the off-target effects shouldn't matter.
@@Flynnbojangels The edited cells go back into the body, along with any undesirable changes.
@@b.a.erlebacher1139but because it’s done outside the body, they can run tests to make sure that only the target gene has been altered, and make any necessary changes if there are any errors, so there shouldn’t be errors being introduced if they do it right.
@@Rwdphotos all tests have a confirmation error, no method is fool-proof
@@Rwdphotos that's probably why it takes 6 months to get the edited stem cells back in the patient. To grow enough of the corrected cells and minimal sick/off targeted cells. You should know where most potential off targeted sites occur but I don't know if there is a 100 percent certainty of it not cutting somewhere else in the sequence
Well researched, executed, and cited presentation. This is a fantastic video and I think the best of what this channel has to offer. Keep up the good work!
Love this guy! Making science fun, not that it ever wasn't, again. Reid is very easy on the eyes and always something interesting to talk about. Keep up the great work. Patron here I come ...
Crazy how the first time I hear about what may be one of the most important developments in medicine in recent times is in a scishow video
It is really, really awesome that we finally have something against sickle cell.
So cool seeing people in the comments who have worked on this ❤
HOORAY!!!!!!!!!! My cousin has cystinosis, we've been waiting for years for this! Fingers-crossed they figure it out for more diseases soon!
What's the point unless you have $3 million lying in bank for cure.
@@Enigma0071 New treatments are nearly always insanely expensive. In the future, prices will drop.
I was wishing for something like this around 40 years ago when my son was diagnosed with "Bubble Boy disease" and we wouldn't have had to go through the Bone Marrow Transplant at Texas Children's.
Be thankful you found a donor, a luxury most people never have
I can imagine parenting a child through a bone marrow transplant. I hope he is doing well.
@@maggierobertson2962 His body rejected the Transplant but it caused his system to start working a little. If they would do a second Transplant they would have to give him chemo and completely wipe out the immunity he had and we didn't want to do that. He lived until he was 18 but he had to get IV gamma globulin every 3 weeks the rest of his life. He didn't really have a "normal" life which is what he really wanted.
@@thug0071984 I was the donor.
@@robinsmith5442 It truly is a shame things like this happen to people. Hopefully we keep findings ways to get rid of more diseases.
Wow, this is a great episode! I especially like how you described the treatment and mentioned the cost!
I had no idea how gene editing was done on an existing person, but I learned something yay!
Also yay Reid 😊
Thanks for making this video. The most interesting part for me was learning how the actual gene editing is done outside the body. It helps me to understand that there are fewer risks than turning some nightmare gene modifier loose in a patient's body.
Incredible news! I'm amazed at how low risk it is from a layman's perspective. As far as the issue is with the benefits not carrying over to eggs and sperm, widely accessible prenatal DNA testing like in Iceland could help parents ensure their baby doesn't have Sickle Cell or other genetic disorders.
The therapy not affecting eggs and sperm is a feature, not a bug in this instance. Modification of gametes (egg and sperm) is hotly debated ethical issue. The general consensus is that the risk of off target modification (like where he mentioned DNA incorporating anywhere in the genome and potentially breaking something) is regarded as still too high with crispr. Essentially, the risk to a potential person who cannot consent to that risk is just too high for gamete modification. Beyond that, the fears that such a modification will open up the door to designer babies is something that is discussed frequently. While it is extremely rare to find people who would support this, changing regulation of gene therapies to allow changes that can be passed to offspring will, as he said, open the floodgates, and not all of the people who take advantage of that will be looking to just change genes to cure future babies.
Your point about preimplantation/prenatal genetic diagnosis(or preimplantation genetic testing) (PGD or PGT) is hugely important. There are still ethical concerns that genetic counselors have to account for, such as people wanting to implant only children of a certain sex or with certain desirable traits, but the potential for PGD to massively reduce the risk of passing on genetic disorders to ones children can have huge benefits. This is especially true as, for many disorders, parents are not guaranteed to pass on a disease to their children. Again, there is a lot of sensitive discussions that have to be had about what traits are okay to select for against, and how do we as a society treat these genetic disorders in such a way that we do not devalue the people who have already been born who have those traits. I am still hopeful such technology can help us achieve a healthier happier future for our children.
Well I’m sure there’s still a decent amount of risk, considering the chemo therapy would wreck havoc on the immune system for a while, as well as impact organs if not enough new blood cells are able to be made by the body. They would likely need to be infused with blood during the entire process and be immunocompromised, so any infection would be an incredibly serious event.
@@brianchew2565Thank you for breaking down such a broad set of issues into only 2 paragraphs - I read it all. The ethical issues are definitely something I was aware of, but you elaborated on some finer points that I needed to hear. I think it's paramount that if society does go down the path of widescale prenatal testing, we make it clear that people still living with disorders - and the qualities of their lives - are still valued.
@@brianchew2565I hear the slippery slope argument, but I can't help but see it as an expensive and physically intensive cure that each generation would have to undergo separately
@@brianchew2565 Good summary! For me, the biggest ethical issue is access. These technologies are pro-human only to the extent that all humans can access them.
So glad to hear! I was so shocked to hear average life expectancy of people with sickle cell was 35yo
Now do an episode about how MDMA/psilocybin are being approved by the FDA for all kinds of treatments. In Australia they’re already legalized for certain treatments of PTSD/end of life depression due to cancer.
Seconding this!! I happen to know someone who was involved in one of the most recent studies about it and it's unbelievably exciting news. It could be such a massive breakthrough in PTSD treatment
i heard about psilocybin being effective for cluster headaches and migraine disorders, i get brain stem migraines with aura i think hemiplegic migraine facial migraines(in the area around your eyes and nose it feels like it’s coming from your sinuses) vestibular migraine and neurological issues from multiple sclerosis including cluster headaches and trigeminal and occipital neuralgia and conventional treatment like nsaids don’t really work well for me so i’m genuinely curious
How wonderful! I hope there's exponential growth and acceptance in this field.
Unbelievable how fast gene therapy is developing!🙌🏼😱
Very believable.
It only took many decades of effort!
more like unbelievable how slow it is, the first genome editing techniques were invented in the 80s, bioethicists and other clueless groups of bureaucratic morons have been holding the field back
Not very fast
Check out the video of a guy learning how to gene therapy his lactose intolerance away, it worked
Incredible. This is the beginning of something incredible beyond what we can even begin to imagine. I’m so excited to see this technology progress and continue to save and change lives for the better. Yay science!
The viral vector treatment is freaking amazing. It's already been approved for my disease, dystrophic epidermolysis bullosa, and there are trials for genetic disorders causing blindness, deafness and many many other severe disabilities. Treatments for all of these are just around the corner.
Hi, it’s a shame that it’s likely to be so expensive in the U.S. and hence likely to be unfeasible for the majority. Similarly here in the UK NICE decides if the NHS will be able to offer expensive treatments. I’m not sure what the answer to this is but hearing of exciting new treatments for some awful diseases is always tempered by the reality of cost smacking me in the face. Hopefully I am wrong and it is affordable for everyone.
My insurance company was more excited than I was to cover my $25k a week genetic treatment. I have theories as to why, but, what it comes down to, is in both the UK and the United States it is not the rare diseases which are causing our health systems to be financially insolvent.
In this case, its a result of the actual cost of development. Developing a therapeutic like this already has massive costs to a company, that they would like to recoup from patients so they further develop new therapies, but the technology to carry out this therapy is also very expensive, and the cost to maintain the standards for a lab to do this and ensure the therapy is safe for a patient is also hugely expensive. At the moment, that means that most gene therapies or stem cell therapies will be wildly expensive. This leads to a lot of discussion among researchers about what therapies are best to pursue, as we want to help as many people impacted by diseases or disorders as we can, but further development of this technology also has a high likelihood of decreasing the cost of future therapies.
All of this is to say that is sucks that this is so expensive at the moment, but there is great hope that costs will decrease in the future, and scientists who develop these (at least within public sector research) are actively looking at ways to make these more widely available all the time.
Searching for affordable alternatives is a big part of science research in general. It's expensive right now, but going by past experience, that will definitely change in the future. E.g. the cost of sequencing a genome nowadays is a drop in the bucket compared to how mind-numbingly expensive it used to be at the advent of that technology
Great video! I love that you went into details of how clinical trials work and pros and cons as well as the basic information about how these techniques work. I can't wait to show this video to my intro bio students! 1 tiny mistake I noticed: Early in the video, the 2 alleles for the hemoglobin gene were shown on sister chromatids of 1 chromosome, instead of on homologous chromosomes.
Chemo/stem cell transplant part isnt as bad as it may sound for most, I didnt have any adjustments to my stem cells but I went through the filter/chemo process to treat lymphoma cancer. Not going overly into detail to keep it shorter, the collection of stem cells takes anywhere from one to several 5 hour hospital visits, for the vast majority its 1 or two days and you go home immediately after they are done. The chemotherapy requires a significantly longer hospital visit, but thats because you lose your immune system and by staying at the hospital you can isolate yourself. Assuming you dont catch anything the other main side effect is fatigue, you will have very little energy immediately after and some fatigue/weaker immune system issues for several months afterwards, but not so bad you cant be at home.
This video's structure and the presentation is very well done. Great job everyone please keep this level of work up! 👍👍 I absolutely love it 🥰
All these major breakthroughs mean is that it will be heavily monetized and then offered to only the smallest subset of people who have immense wealth and resources
This is either the first or second time in my life that I've heard medical progress actually making it patients! Keep the good news coming!
Clearest description I've heard. Thanks.
I was in girl scouts with a girl who had sickle cell disease (at the time i knew it as sickle cell anemia). I recall her going through a BMT treatment. I haven't seen her in probably 20 years but i hope she's doing well.
I worked on this :)
As a patient for a drug that you likely didn't work on (Vyjuvek), but also uses some of the same technology, thank you so much. You're changing lives.
Congratulations
Congratulations!! :D
Thanks for this, its extremely rare to hear firsthand from someone who is directly benefitting from our work, when working in a lab/cleanroom i find we become disjointed with the end goal and this means a lot!
When are you going to use KRISPER to edit out viruses like it was made for like HIV, HPV and Covid19?
Sickle cell is prevalent in areas with high Malaria burdens since Sickle cell disease confers some resistance to the Plasmodium parasites that cause Malaria.
I’d be curious to know if this treatment can remove the negative effects of the disease without removing the resistance to Malaria. If the treatment does cause a reduction in Malaria resistance, it could cause more harm than good if deployed widely in Malaria endemic areas without additional Malaria mitigation work.
I'd imagine most of the people who could afford this can afford antimalarials
@@CaTastrophy427 I imagine most of the people who can afford this don’t live in places where it matters.
It will matter if mass deployment ever becomes possible, which seems probable over time.
0:22 you underestimate my hyperfixation on CRISPR
"CRISPR-Cas9, Bring me a Gene..." 😁 -- Acapella Science
@@matthewcox7985I was singing it in my head the whole time!
@@matthewcox7985W reference
Indeed!
It's important to remember that tech of ANY kind is ridiculously expensive at first, but as the tech gets refined, the price comes down. These treatments are in the millions currently, but I expect in 5-10 years it will be in range of us commoners.
Respect for all the Geneiuses working on the cutting edge for Humanity's Crispy future.
amazing!! this is the best news i've heard in a long while
I would love to see you cover how Vyjuvek from Krystal Biotech works for EB. It is a virus vector treatment, the first topical ever approved. I have DDEB and it "helps," but it is life changing for RDEB.
I desperately hope the cost changes soon. A cure for chronic disease like this is miraculous... but if that cure can be withheld so easily, the shadow of eugenics always looms. I'm sure there's going to be significant research about making CRISPR treatments more cost effective soon, so here's hoping.
Oh God...yet another reason this next election will decide the fate of the world...
I’ve been waiting for this day! And the trails will soon be practice!
Family member had a blood disorder and had to get a bone marrow transplant. This seems effectively the same process but instead of a donor donating stem cells, the patient's own stem cells are used. This is good though for not having to worry about rejection. GVHD & chemo were the worse parts. This removes one from the equation.
I did one of my bio presentations on CRISPR years ago. I’m glad it’s starting to get the attention it deserves. 😊
I really hope this could one day cure my genetic disorder
They haven't cured mine, but the viral vector treatment was developed and it does work as a treatment for my genetic disease (Dystrophic Epidermolysis Bullosa). I hope they cure yours.
@@edwardallenthree mine is for Ehlers Dan Danlose syndrome. I have absolutely no idea if they have even tried or started on it yet.
Many years ago we had a young African-American male come into the hospital in respiratory crisis form a Colorado mountain town. Ironically (as you will see in a minute) he was flown in Flight for Life. It was determined that he was in sickle cell crisis secondary to altitude. The recovery hospital was at an altitude of over 6000 feet and he was stabilized there. Once he was well enough to leave he was told he could NOT go get his car and that he needed to take a VERY long way home avoiding all mountain passes. I've often wondered how he was doing.
With regards to sickle cell. It appears to be concentrated in Certain parts of Sub-Saharan Africa. With Malaria being an issue, this would make sense. either the Malaria gets you, or the Anemia. I am wondering if there are different health outcomes between those who live a more indigenous lifestyle (local foods/medicines/herbs/work habits/sleep habits) who have sickle-cell compared to those afflicted who live in Urban environments.
The fact that Heterozygotes of the sickle cell disease persist to this day show that it was slightly selected for in terms of population genetics. Those who could resist malaria survived and were able to pass on their genes. This explains why we see it more in these populations, but having both copies of the gene is still detrimental no matter the lifestyle. Without access to treatment, I'm sure having sickle cell is life threatening, it's just a matter of time.
@@billybob7688453 Im curious about the local herbs, remedies used. Ive been reading up on different non-western medicines. this is just another one to add to the list :)
All great things start with humble beginnings, Crisper might only be helping a handful of people right now but soon it will be helping the whole world.
My conclusion is that this CRISPR-CAS9 treatment shares a lot of similarities with bone marrow transplantation of a matching donor. However, the donor is the patient itself this way. There are a few creases to iron out, but I believe this has a great future.
A sat in on a talk about CRISPR back in 2022. Glad to see this kind of progress happening!
Learnt about fetal haemoglobin 2 weeks ago when looking up the haemoglobin gene. Good to know it's still useful in adults.
I have a blood disorder (von Willebrands disease) so this is great news. I can imagine the utility of CRISPR will expand pretty rapidly now that it’s already being used clinically.
Thinking about it.. I *did* hear about Crispr 10 years ago. I always wondered why they didn't deploy it sooner! I know testing is important but this can change so much for the better!! :D
As someone who had 2 different bone marrow transplants (for myeloma [plasma cell cancer], but same process), it's always weird to see what else that process can be used to treat. The process is pretty rough, and when a donor is involved the long lasting side effects (GVHD) can be rough too, but when the alternative is worse, it's still worth it.
Hopefully we come up with a safe way to do the gene editing inside the body instead of having to do the whole transplant thing.
Well it's progress, but it sounds like it is still an extremely arduous treatment. A significant advancement, but probably doesn't open the floodgates to use CRISPR for all sorts of problems.
Science has already saved my girlfriend with CF, now it is saving more people. Im so thankful for all the work being done across the field.
The price tag really saddens me a someone with the trait and who has a cousin with the full disease 😭 hopefully the price comes down soon
The price tag is for all of us, not you. That is why we have society. I will gladly pay more in insurance or taxes to cover you, and I hope you would extend me the same courtesy.
I was shocked when my insurance covered a genetic treatment that is similarly expensive for me (25k/week ongoing treatment). I felt guilty. One of my docs said, "don't feel guilty about how much insurance pays on your behalf." Another said, "you are an extraordinary person with an extraordinary disease that requires extraordinary treatment, don't feel bad about people helping you." That having been said, if I can skip a treatment, I do.
Baby blood instructions located different than where adult blood instructions are. Is that so the baby has a more likely chance to match the mom's blood type and antigens on her blood?
1:58 The story of the adaptation/mutation of hemoglobin beta to protect against the billions of historic deaths from malaria should not be wholly divorced from the story of sickle cell disease and its treatment: the gene is a trade-off, not a defect.
Very exciting. Also very sad knowing there is a cure for something, but few can afford. Hopefully in the years to come it will become more affordable so the people affected by this disease are able to get their lives back.
I'm going to venture to say that any American who wants to pursue this treatment will almost certainly find whatever funding they need, and I say that from experience of having a rare disease and needing funding to cover a new genetic drug treatment.
The problem with these treatments at this time is not their expense. It's that, right now, so few people actually qualify to get it.
*great news!* though imo, _in situ_ and germline editing should be allowed soon, assuming good research outcomes.
Wow, this prospective technology sent me down the route of becoming a scientist a decade ago - and now, working in clinical immunohematology, it’s more exciting than I can put into words knowing that some of my sickle patients may soon be able to stop coming in for their exchange transfusions!!
For starters we have other sophisticated new treatments that needs less intrusion than this but since these treatments well studied and got enough trials they can get pass. But once floodgates open we can see more sophisticated cell and tissue targeted gene therapies without chemos or lengty processes in the field. In medicine tech always go way ahead than the aproved trwatments due ro lengthy process of getting it aproved
I really wish I could get involved in a company that is doing this kind of research, but I just have a background in data science and nothing in biology.
I've been researching everything we know about crispr since i was eleven, i thought it may be able to help with my mom's rheumatoid arthritis (it might be possible), but now more than ever since I've learned i have multiple genetic chronic conditions including Hypermobile Ehlers Danlos syndrome, MCAS, and a few others, i am excited to see how it progresses
Similar situation here. I've been waiting for news on CRISPR treatments since it came out!
I wonder if this could be useful for Hereditary Spherocytosis (Sphere shaped red blood cells). My mom, uncle, and grandfather have it.
I love that f*cking shirt. Where can I get one? Oh, and yes, CRISPR is an awesome look into our future. Keep pumping out these great videos. Love yous at SciShow. Seriously about the shirt bro.
12:11 In this case, there is no way around it being so expensive. It is the cutting edge of medical technology and that is going to cost a lot of money. It has to start somewhere and some people are going to pay through the nose to have this treatment, which will make it cheaper over time.
There will be a machine that can take over the laborious manual tasks and cut a lot of cost, and probably time.
Treatment being expensive is the norm in medical science, as is the price coming down.
I mean, yes, BUT the question of who foots the bill is incredibly relevant. Some countries force individuals to pay a la carte for treatments, other countries socialize all/most healthcare costs. So, expensive doesn't have to mean unaffordable by default. The outcome for individuals is very sensitive to the economics of healthcare in a given country.
There is a common mistake in this video. Both chromatids of the chromosome sketched at 1:53 have the same identical copies of any given gene. If there is a different copy it is located on the other chromosome of the pair.
I have heard it from an episode of the Blacklist !
I have one copy of the sickle cell gene. When my mom married my dad, they had tests done on him to ensure there was no chance he'd have another copy of the gene, as to avoid children having it. It's nice to know the next generations of my family will not have to care that much about this anymore
I am a lab tech that makes the Casgevy product. It's extremely labour intensive and not really open to automation, so I'm afraid the price isn't going to go down any time soon
Would in Vivo editing solve this problem?
Ok this is pretty off topic but I saw the bell-ringing stock footage and finally realized what that bell for is at my cancer center lol. I go there a lot because it's technically a cancer and hemotology clinic and I have to get fairly regular iron infusions for my anemia.
Holy smokes maybe they can finally cure my Agammaglobulinemia
It sure looks promising!
This was awesome. Keep fighting the good fight!
Really hope that price tag is the economics of scale issue, and not a fundamental cost of the treatment
CRISPR & AI could advance medicine faster than ever before, as information now doubles every 3 yrs its incredible to imagine finding the cures that will end suffering for humanity. Imagine lowering the cost of cures are the norm rather than expensive pharmaceutical treatments. The benefits definitely outweigh the risk.
Grab your frogs, we're making dinosaurs!
I'm glad they didn't shy away from the cost of this treatment. It is eyewatering. And it's even more concerning that there isn't much to be said about how it could be reduced over time. And there's the other question of how cheap you can go without jeopardizing treatment integrity.
We need to put in at least 1-3Mill a year into this because this is the beginning to ending cancer and other problems in the body. This might also go hand in hand with curing aging and gene editing so that we can stay younger much longer. I hope we get a lot more news on this as the years go on and maybe get a yearly update on it’s progress.
*"To Me, My X-Men!!!"*
Wednesday
Maybe one day it could help people with my condition, too. It’s exciting! I hope everyone who is having this treatment live long, happy, fulfilling lives with no complications from their original disease or the treatment!
Killing off all your remaining stem cells intentionally sounds wild though
This made me cry, this will help so many people.
So nice to hear how the wealthy people are benefiting from modern medicine. Warms my heart.