MECP2 Duplication Syndrome is Reversible!!! - The 401 Project | Cure MECP2
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- Опубліковано 6 жов 2024
- Director: Joseph Mendoza
DP: Nick Schwyter
Editor: Ed Ng
Today, November 25, 2015 - the world heard the incredible news that Dr. Huda Zoghbi and her team have reversed MECP2 Duplication Syndrome in adult symptomatic mice using antisense oligonucleotide (ASO) gene therapy!!
This groundbreaking research shows that ASO gene therapy is a promising approach to treat MECP2 Duplication Syndrome.
We are very optimistic that a cure is on the horizon for MECP2 Duplication Syndrome, and it's sister syndrome, Rett Syndrome.
We still need to fund research to get us to clinical trials. Please donate to the 401 Project to make this a reality for our children. The power of research was proven today. www.401project....
Thank you!
We Will Not Give Up!!!!!
This is an amazing achievement, I wished my brother Ashley who died 27 Feb 2016, aged 14 from adenovirus + secondary duplicate syndrome was around to receive a miracle rather than be treated with meds for presenting symptoms. This is a very rare genetic disorder occurring higher in boys and the symptoms get worse as they age. He had seizures, he couldn't walk nor talk, he constantly required 24/7 care and had continued pneumonia. I hope more can be done to help eradicate and help those presently diagnosed with MECP2 see a better tomorrow, everybody deserves a wonderful life.
Wow. The idea that we could do this is really exciting! We live in the future.
Great work!!! How does one send any help possible. Please include a gofund link or something. We need to make this go viral and raise awareness and alarm to the world!!!! Exciting stuff!!!! Hope all children can live to see the day this comes to fruition.
Thank you for explaining with clarity what this important milestone is all about. Today is a great day.
Amazing!
Very exciting! Fantastic!
Has the research gotten any closer to testing on primates. My grandson Gavin turned two in Sept of 2018 We are all praying for a cure for Mecp2DS
Go on the mecp2 duplication syndrome family talk facebook group for support and questions, you'll know all the latest news
Every new day we hope to return our son to normal life, everyone 😔
Please help my nephew 💔😔 hes 4years old in NZ!
MECP2 is not reversible in humans. The testing was done on mice. The headline to this video, and the other videos you posted regarding this, Joseph Mendoza, is misleading. You really should change the title to reflect it is reversible in mice.
you know what you can only pray for healing thats what you can do. stay blessed.
The MECP2 Registry is a participant-driven project, overseen by MDS research advocates, with the purpose of furthering our understanding of this disorder and developing novel therapies. Please register at
mds.nrihub.org/#/main