Can CRISPR cure Sickle-cell Disease?
Вставка
- Опубліковано 1 чер 2024
- Sickle-cell disease is one of the most common genetic conditions worldwide, with more than 6 million people living with the disease. Three-quarters of them are in sub-Saharan Africa, where childhood mortality due to sickle cell remains high.
Previously, the only curative treatment was a stem-cell transplant, but new trials are investigating the potential of CRISPR-Cas9 gene editing as a tool to cure this chronic condition.
One such trial has already achieved exciting results. For people around the world who are living with sickle cell, these trials could offer hope that long-term cures are just over the horizon.
Read more about sickle-cell disease www.nature.com/collections/sickle-cell-outlook
This Nature Video is editorially independent. It is produced with third party financial support. Read more about Supported Content here: partnerships.nature.com/comme.... - Наука та технологія
![BREAKTHROUGH: Scientists Reverse Blindness [CRISPR Technology]](http://i.ytimg.com/vi/rbVvu_104Pg/mqdefault.jpg)
My academic advisor spent years developing a sickle cell disease drug, when CRISPR-Cas9 gene editing was discovered and sickle cell disease gene editing therapeutics were becoming increasingly plausible, he didn't care if years of his work would become obsolete. He just cared about sickle cell patients having something to assuage their pain.
Still probably worth while to look through the pharmaceutical approach to getting hemoglobin to fold properly
@@sagestrings869 Oh, and it's always worthwhile to explore pathways like this. Even if the science doesn't work out, you at least can try publishing so other geeks know it may be better not to pursue the same path
@@alexplastow9496 Also, the knowledge gained may be useful in other research.
Please I'm a sickle cell patient from Nigeria I need help please help me
🙏 prayers to you all who are living with this illness. My beautiful strong wife has sickle cell and Its so heartbreaking to see her feel this pain and the doctors and nurses treat her with less than acceptable care. I won’t let this continue I’m learning to advocate for her thank you for the information of the illness. Goals to get all sickle cell patients cured of this horrible disease
Aww as a sickle celler, I say thank you for this. Such kind words
Sickle cell has ruined my life rather hard. I tried joining the Air Force and I found out I had a 48% hemoglobin S type. I was trying to stay in and they still kicked me. After that, my life has been a shitty downward spiral and I'm fortunate to be 40 years old now with this damn thing feeling like it's trying to kill me every SINGLE F'n day!
Your wife is fortunate to have you bro.
Thank you 🙏🏾
❤
@@magus7246 thank you 🙏 I believe I am the one who is fortunate to have her. And I’m sorry to hear this we will be praying for you m. Have you looked into the gene editing? It’s had a 99% effective cure rate for sickle cell patients. The damage sickle cell has already done can’t be fixed but the people who have been cured have even made it to point of not having to have pain medication or any hospital admissions. Definitely something to look into and see. We are currently looking into this come hell or high water if I have to sell everything we have to get it done we will. So there is hope don’t give up.
I can't wait for the day this is accessible. This will also make alot of couples with sickle cell trait very happy, knowing that they can have children without worrying about SCD.
praying this works in my lifetime. Im only 14 and i have been in pain almost every day of my life. I really dont want to die young due to this disease i did nothing to earn. Praying for myself rn
My mother has the disease and she’s been fighting it every since she was a kid, she’s now 46 and still going on strong because she dedicates most of her time taking care of her body, making it to her appointments and taking her meds everyday.
I literally HATE that i have to see her struggle thru this ever lasting long pain that she manages everyday, but i also commend and thank god everyday that he gives her the strength she needs she to fight and see a new day and that Hope will prevail not just for my mom sake, but everyone else suffering from this ridiculous sad disease.
My msg to you strictly tho….is to pray and thank god to be blessed to see another day because you may never know…..but one day you may hear about a cure that’s ready and developed for sickle cell.
STAY STRONG
BECAUSE ONLY
THE STRONG SURVIVE🙏🏾🙏🏾
@@Reverb_Lucas AMEN🙏
Thanks, bacterias, for CRISPR!
This is absolutely amazing! I'm glad these treatments are out so quickly after the discovery of Crisper
Hmm... I'm not sure if "quickly" is the right word. CRISPR was discovered in 1987 and different researchers have been making adjustments since. If I'm not mistaken 2013 was the first practical use of CRISPR in a flu vaccine.
I'm not sure but if it's possible I would choose these to cure my disorder.
I am so happy to tell everyone that I totally cured sickle cell anemia with herbs, my mom also cured Athritis after 12 years. Everyone should follow Dr. Ewi1 and tell him about his health problems. He has natural treatment ua-cam.com/channels/YXkCmbhWCpfXIbnBuXCpUQ.html
Is even affordable, and how one book an appointment for the crisper cure or treatment
I lost my high school best friend today due to sickle cell disease 😭😭😭God this hurts
I pray you rest well love 💗 sleep well till we meet again 🥺🥺
am sorry my son die June 1 from sickle cell disease
Yo, I have SC as well (Male).
Been dealing wit it for all my 3 decades of life.
My thoughts go out to yall's Sickle Cell Warriors. 🖤 🙏🏾
What happened btw?
Like, deeper than jus "complications due to Sickle Cell".
Do yall kno what it was exactly?
Cardiac arrest, Organ failure, Infection, etc?
Thnx in adv for both yall's responses.
FDA approved 👌 Let's go SCD community ❤
I'm sufferings from this disorder now I'm glad that treatments are coming but it's really expensive. Hope I will die without sickle cell.
Ready to try anything for my baby...
Life science have many secrets 🔥
CRISPER is such a great discovery amongst all.
Need more work on it to get more helpful output.
Biology is just.. wow
Tell me about i have sickel cell and it sucks
@@gamerjay5869me 2
I am so happy to tell everyone that I totally cured sickle cell anemia with herbs, my mom also cured Athritis after 12 years. Everyone should follow Dr. Ewi1 and tell him about his health problems. He has natural treatment ua-cam.com/channels/YXkCmbhWCpfXIbnBuXCpUQ.html
Bless you. I'm so glad that you were healed! I pray it becomes accessible to everyone that is and need of this great blessing
My sister died of this..rooting for you scientists
God is truly a blessing and watching out after I Sickle Cell patients. Let’s stay strong and work together 💯🙏🏾🤙🏾
This should be just the beginning. There are thousands of other genetic diseases which should be curable with enough government funding. Let's invest in science again and put our tax dollars to get use.
This is surely exciting. CRISPR Cas9 should be used in the right field like this!
Crispr is awesome
Wow I would love to get that done.I was born with sickle cell and I am telling you it hurts like hell
@Ganesh Thakur no I did not
Sickle cell trait should also have this option as we are symptomatic as well.
It wouldn’t completely work for the trait. Also, trait patients would not get priority over SCD patients.
Amazing explanation
Great content
Everyday I wish I could live a life without sickle cell God has blessed me to see 41 years old. I feel so bad for the babies who suffer from this because I know exactly what they are going through. I figure I made it this far might as well ride it out until I’m gone but I’d love to see the day everybody who suffers from this gets a chance to really live life.🙏🏿
Wow, I didn't know about hemoglobin F and I looked it up. Super nice, mother nature is astonishing!
Read about fetal hemoglobin here, towards the end of the document
truly impressive.
What country are you located. Where is ur hospital center please.
Unleash CRISPR in to the hands of the clever & desperate! Let the nerds & determined flex upon the world ❤️
Except that isn't how it works. Global criminals are in charge.
I'm a sickle cell patient from Nigeria I'm going to 17 this year please I need this treatment
It’s just Awesome
How about a go at atopic eczema too?
This is a wonderful therapy to reduce symptoms in patients. But I can't shake the feeling that I'd rather have the underlying mutation cured rather than trying to reduce symptoms.
Of course that's nuanced (in malaria-burdened regions, this might be the better option), but settling for symptomatic rather than causal therapy seems needlessly underambitious.
I mean I suppose in some sense it's just treating a symptom. But in the causal chain of symptoms (genetic defect - > error in the production of adult hemoglobin -> misshapen red blood cells -> diverse set of symptoms including pain, organ damage, etc) it's pretty far upstream. And remember, genes are in fact being changed here. Genes in stem cells in the bone marrow are being altered to produce healthy red blood cells.
The logic of this cure is as good as fixing the root cause since it allows the body to produce what it needs. When you need to turn left it doesn't matter which of your hands is holding the wheel.
BEAM therapeutics is starting trials in early 2022 to do exactly that. They have a much more specific crispr editor that can do base pair switches and make more specific edits to actually fix the root of the problem.
After suffering from herpes for a very long time, i came across Dr ogbebor herbal centre on UA-cam and he cured me with herbal medicine.
It is not treating the symptoms it is treating the disease bc your body only mutate hemoglobin A into sickle blood cell but now it is changing the type of hemoglobin into hemoglobin f which means your body won't be able to change the fetal hemoglobin into the sickle shape.. Then it cures anemia. Cures pain as your body is producing healthy blood cells which allow blood flow properly
Excellent video!!
The main expected problems that may arise as a result of the use of gene therapy and genome editing:
- disorders in the work of the genome;
- effect on gene expression;
- changes in epigenetic regulation;
- genome instability;
- violation of DNA repair processes;
- - mosaicism and tissue chimerization;
- disturbances in protein-protein interaction and in molecular cascades.
As a result of such changes in molecular processes, previously started pathological processes may progress or new ones may be induced. These can be various chronic diseases, diseases of the immune system, metabolic disorders, blood clotting disorders, oncological diseases, rare syndromes, infertility, etc. But the most basic and most anticipated consequence, which actually includes all of the above, will be cancer, premature aging to one degree or another.
Please who can help me here to get the contact to get to this people...I have a love one to save please 🙏🙏🙏😭😭😭
how do we access this treatment?
If a mother expresses fetal hemoglobin after this therapy, might that lead to complications in pregnancy, as there's no difference in oxygen affinity between maternal and fetal Hb anymore?
Are you referring to beta sickle-cell disease or all expressions of the thalassemia which also includes alpha thalassemia Hg-H CS? I know hemoglobin H disease has a high affinity for oxygen but I’m not so sure with Beta Thals major or sickle-cell..
THE PROBLEM IS PEOPLE LIKE ME WITH SICKLE CELL JUST TURNED 40 will probably die of this disease before these cures can be made available to everyone. Sickle cell will be a thing of the past in the future but will some of us also be.
Hey check out A lady named Tamika Mosley and Healing Blends Global on here. They work on natural Sickle cell management and have products she is very accessible and does consultations. Hope this finds you
Are you in the US?
Yes I am
@@RHt09 yes
After suffering from herpes for a very long time, i came across Dr ogbebor herbal centre on UA-cam and he cured me with herbal medicine.
Bom dia Dr.
Por favor tradução em português.
Percebo muito pouco ou nada de inglês.
Don’t give up my friend there’s a permanent solution to your problem visit Doctor Ani John on UA-cam channel he cured mine completely with natural remedies..
I am happy that patient's have light at the end of the tunnel. Bluebird Bio is in the final stages of clinical testing their gene therapy "LOVO-CEL" and CRISPR has a similar gene therapy "EXA-CEL" which hopefully and GOD-willingly can lead to transfusion independence.
Any risks of producing proteins that are ment by evolution to stop being produced after birth? Is there any significant difference between fetal haemoglobin and adult one? Anyways… if this is helping people without having any colateral problem we should continue in that direction. Great job!
Fetal Hemoglobin binds oxygen a bit better. This ensures that the fetus always gets all the oxygen it needs.
This also means that fetal Hemoglobin would hold on to this oxygen a bit more, not allowing the tissues to take it up as much. This might mean that individuals with fetal hemoglobin feel the effects of hypoxia a tiny bit earlier on a cellular level (complete speculation on my part). At normal oxygen levels it would make no difference though.
Please how much does it cost?
How can be cure with crispr and how does it cost
Finally found a cure for my son Sickle cell I’m so glad you came into my life. I pray for long life so you can save more souls,Thanks #druromi
I'm reading Jennifer Doudna's recent book with Samuel Sternberg. I intend to do a research on gene editing in my undergrad or after. Which course would be the best to start? Any suggestion?
I suppose any course on molecular biology and genetics would be useful. Also general cell biology can also be helpful because genetics actually involves quite a bit of transporting and processing all around the cell.
There are two ways to do so. 1. In wet lab by joining any cell and molecular biology course? 2. Dry lab like working on bioinformatics, Genomics and Proteomics etc. All the very best. You are already made a great decision.
After suffering from herpes for a very long time, i came across Dr ogbebor herbal centre on UA-cam and he cured me with herbal medicine.
I have a question, why DO the cells switch off the production of fetal hemoglobin?
Fetal hemoglobin binds oxygen a bit better than adult hemoglobin does. This ensures that when the blood of the mother gets to the fetus, the oxygen gets transferred to the fetal blood, rather than staying in the mother's blood. When you're not a fetus anymore, you don't need this tiny boost in oxygen affinity anymore and if you're a woman it's actually important that your hemoglobin binds oxygen less good so that if you get pregnant, your fetus can get your oxygen again.
@@Pyriphlegeton ohhh, that clears it uup, thank you
@@Pyriphlegeton
Thanks.
Great question. Im also thinking there must be a good reason for this. Another concern is, what are the negative consequences of flipping back on this switch?
After suffering from herpes for a very long time, i came across Dr ogbebor herbal centre on UA-cam and he cured me with herbal medicine.
Comment for the algorithm!
I was born with SCD and I'm 33yrs old Now. However, I haven't been having severe crisis, frequenting the hospital, taking any medications or having transfusions. This keeps me wondering why they say I have the disease and why I'm different from others with this disease. I've always had questions.
My family and friends doubt that I have it which made me doubt that I have it too, but I feel the fatigue, sometimes with the yellowish eyes, low Hb counts, one time severe pain in my arm after exposure to cold wind and lately with migraines (started 2yrs ago) which I have been controlling with supplements and hydroxyurea. I've been on Hydro for a year and 5 months now but I still get the migraines sometimes and for the past 4-5 weeks, I've been battling with it everyday of my life. They said my brain doesn't get well oxygenation due to the sickle and inability to transport enough blood to the brain. Also, another doctor saybi need to stop the hydroxyurea because I'm a lady and would want to have children someday and this med affects pregnancy. He says they don't recommend it for ladies over 20yrs old. I'm considering stopping it and staying on just supplements.
I have once been told by a professor that my body probably produces more of fetal hemoglobin but this wasn't diagnosed, it was just a mere guess. After watching this video, I think it might actually be true and I'm willing to go through any tests with any doctor or researcher on this. I'm tired of guessing and asking questions, I want answers. By the way, just last month, I had the highest Hb count which my doctor told me that is so good and no blood transfusion is recommended even for people with normal blood count; at the time of that test and the doctor was saying that, I was battling with migraine and this added to my confusion.
If there's anyone with answers, suggestions or the kind of tests I need to go for, please do. Thanks to everyone on this research.
Chances are you probably have a lower 'type' of Sickle Cell; the disease has its own spectrum. Those who are constantly bed-ridden in hospitals and need regular blood transfusions are Hemoglobin SS, the most severe type. I personally have Hemoglobin SC (the second-worst behind SS) and I've had a fair amount of crises at 27 years old, but none of them were life-threatening thankfully and I've not had to get any blood transfusions. Here's hoping it stays that way lol
Have you been officially diagnosed by a doctor that you have it? I've know I had sickle cell my whole life because they tested me as a baby.
I recently got a confirmatory diagnosis and it still says HbSS. This is the most confusing part. I mean, I'm thankful I don't have severe crisis and blood transfusions but I'm just curious.
@@cr8444 If you really have HbSS, then consider yourself very lucky lol. Whatever you're doing with your diet/self-care, don't change it 😂
Could you share your diet?
I really need this treatment for my lovely son as soon as possible. Were do I go?
You're not the only one who's asking this, sadly the person who made the video doesn't respond back to the viewers like you who're asking for help. I guess you can asked your doctor about it, but also i'll be careful about tampering with your genes it sounds good an all but, at the same time im suspicious. It's been around since the 80s and still little as been known about it.
By the way where in the world are you located? Try letting your little one do some deep breathing excersie to get oxygen to the brain use burdock root? elder berries boil them as tea and give it to your child no sugar, it helps to clence the blood. Also lots of berries, like strawberries black berries blue berries etc put them on an all raw food diet like fruit juice etc is best in helping with his or her health.
Here is a brother here on UA-cam who can help your child to get rid of the disease Name Yahkiawakened website is www. yahkiawakened.com check him out he as all the herbs you'll need to help your child.
I'm not pointing you in the wrong direction this man is legit certified chemist herbal healer etc, anymore question about him I'm here to help. Sometimes we need to go back to nature for our healing these doctors and pharmaceutical companies are ony it it for the money and nothing else good luck hope you followed my advice
You can contact me.
Please where do we go to get the gene mutation treatment please. What hospital please and I. Which country is the hospital located please@@NoRelig._Lawful_Free_NoPolit.6
Gene therapy is widely advertised, and various information resources are being created demonstrating the benefits of genetic screenings, perinatal diagnostics, genetic passports, etc.
The most surprising thing is that at such events the emphasis is on monogenic diseases and specialists are invited to share their experience exclusively with regard to the treatment of individual patients, without going into theory and generalizations.
A clinical case report or a history of a single disease can undoubtedly be very interesting and instructive, and there are many scientific publications in medical journals devoted to the medical history of one or more patients. But the deliberate exclusion of theory and generalizations from reports reminds me of the manipulative technique of shifting the angle of attention.
The topic of gene therapy spreads gently and imperceptibly, as if it were just a new generation of pharmaceuticals. Nowhere is it particularly advertised that this is a new experimental approach, with its "pitfalls", but it is advertised as high technology.
So what's make it that much expensive?
Because you have to be admitted to the hospital, have your stems cells apheresed out of your body, genetically edited and infused back into you after undergoing chemotherapy and then watched in the hospital for a significant period of time. Cure will likely be between $1 to $1.5 million per person and be heavily subsidized by government.
It's called capitalism.
@@jinndoe7068 Yes, these discoveries, the hundreds of millions of private funding necessary to develop them, test them, scale them up to commercial scale, get through multiple stages of FDA approval, multiple stages of human testing are all because of capitalism. Communism and socialism create nothing of value and offer nothing of value to society.
After suffering from herpes for a very long time, i came across Dr ogbebor herbal centre on UA-cam and he cured me with herbal medicine.
I am also suffering from sicle cell disease..... I want to know .....where this man get rid of that disease..... Pls someone tell me....this is my last hope..
You can contact me.
This aged well. Now I just need to find $2 million to pay for it 😂
38 years of my life
When this method available and affordable in Indonesia? Please help. 🙏🙏
How can we contact you?
"It should be Global."
If only more people were hurt by disease, perhaps they would hold the same values as you and I.
Please can some one help me what is the name and how can I get access to this hospital please I need help.
Please help am always in crisis,and am in Africa,it hurts
Hello sir.
Hope my message can be sent to you not so lately.
You can contact me.
i have achild who is known sickle cee patient at 10 /12.how much can that procedure cost and where can it be done
You can contact me.
Meeting you DOCTOR OKUNABA on UA-cam is like meeting GOD himself because, what you did in my life is what no other doctor can do. Thank you for DOCTOR OKUNABA for helping me Eradicate my HSV1 & 2 naturally, you are a life saver may God bless you
Please I need help, my partner and I both have the sickle cell trait, we need help
@@ndiannabel6315 You can contact my email in my channel
How do I access this option for my 7 year old. My heart breaks everytime she breaks down. 4 admission in 2months.I just want her to be free.
You can contact me.
I’m praying this is a great discovery , my son died 4 years ago , I don’t wish this got no one .
For CRISPR to become successful, humanity will need a few guinea pigs to help improve the science.
me )
Omg! Listen to what you are saying.
Am in Uganda, and how can I get it coz my son of 1year and 4 months was born with the disease. Please I need your help.
Hello madam. Hope my message can be sent to you not so lately.
You can contact me.
How can I get access to this treatment?
You can contact me.
Not shaking their pockets 😂😂😂 I keep pain meds on me 😂
I am all for.curing and treating genetic.diseases use crisper just don't abuse it
I wish I knew this for my daughter who passed away june 2 2022.
Crispr👍👍👍
sicko mode
6:25
Guy who made aids says good luck
🙏❤️X
homeopathic medicines can help to reduce this type of problem or cure. Vanadium met etc. lot of dilutions can be used.
TL;DW: It's possible but only for the rich
As every technology is at first.
Computers less capable than any smartphone were multi-million-dollar projects once. Now they're common even in poor countries.
@@Pyriphlegeton I guess this _tends_ to be the case... but they didn't _have_ to feature a patient using a roughly 1000$ phone. Tasteless... saddening...
“The future is already here - it's just not evenly distributed.
― William Gibson, The Economist, December 4, 2003”
user73o1u 81716 this poor guy has been dying of pain every day of his life and has been basically disabled and without home and you focus on his cell phone. You’re disgusting.
@@RHt09 with all due respect, focusing on wealth inequality, especially concerning health care, seems more important than ever. But once you notice it's indeed, disgusting, and may surpass an individual's suffering
How about men & women who carry the trait just don't have children together? This disease is 100% preventable...what am I missing here?
Unfortunately these drugs are over a million Rands. I work in a hospital in a country where sickle cell anemia is endemic... no one will be able to afford this therapy here. Almost useless actually.
Pls how can I contact you ,or chatt with you
Am so happy right now am Herpes negative thanks #drodukuhome ❤️❤️