RSRT

@davidrliu So exciting! Congratulations to all. Keep going! Patients are waiting.

In a major milestone for science, for medicine, and for patients, the world’s first CRISPR drug has been approved in the UK. “A new treatment for sickle-cell disease and transfusion-dependent β-thalassemia has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 12 and over after a rigorous assessment of its safety, quality and effectiveness.” Congratulations to the many scientists, doctors, patients, and industry leaders who made this development possible.🍻 gov.uk/government/new…

Help advance a cure for Rett by participating in the Rett Syndrome Global Registry. rettglobalregistryx.acrossmatrix.com/en-US/#/user-r…

Taysha has shared a community letter regarding their clinical trials in Canada, US and UK. reverserett.org/news/articles/…

@TayshaGTx reports on safety & efficacy for Patient #1 in Rett gene therapy trial. reverserett.org/news/articles/…

@TayshaGTx reports that Patient #1 in their gene therapy trial has improved! reverserett.org/news/articles/…

@UrnovFyodor I am saddened to hear this news. I met Don through the Life Sciences Research Foundation and admired his great passion to help young scientists.

Along with all other gene editors I am indebted to Don Brown for his pioneering work on zinc finger proteins that enabled the first gene editing wave. On a personal note, I trained with Alan Wolffe - who trained with Don. Don's was an inspiring and impactful life.

We've awarded additional funding to Pete Beal to advance an RNA editing project. This genetic-based approach aims to correct the mutations that cause Rett by harnessing a natural protein already in cells. reverserett.org/news/articles/…

Our CEO Monica Coenraads was recently interviewed on a Turn Autism Around Podcast. Watch it for a thorough summary of what causes Rett and the current state of the research. marybarbera.com/rett-syndrome-…

Taysha announces that they are cleared to dose Patient #2. Update on Patient #1 will be shared mid-August. reverserett.org/news/articles/…

See Rett syndrome through Emerald eyes! Our latest blog by our CEO, Monica Coenraads, shows how Emerald quantifies symptoms in ways humans simply can’t. reverserett.org/news/articles/…

Neurogene's CEO, Dr. Rachel McMinn, provides details on their Rett gene therapy trial. A short registration form is required to access the webcast. edge.media-server.com/mmc/p/q3vx354g…

Congratulations to everyone at @NeurogeneInc and @neoleukin

With Emerald, doing nothing is changing everything. Dina Kitabi explains how her sci fi tech of the future is revolutionizing Parkinson’s. With RSRT funding we’re excited about what Emerald can do for Rett. youtube.com/watch?v=TLxiiJ…

RSRT is laser-focused on genetic medicines. Gene therapy is a genetic medicine that delivers healthy genes to compensate for mutated ones. Learn more about this approach with this short explainer from the American Society of Gene and Cell Therapy. patienteducation.asgct.org/gene-therapy-1…

We aren't the only species that edits our RNA. Check out what octopuses can do! npr.org/2023/06/08/118…

Happy 4th of July to everyone celebrating!

CRISPR is not the only genome editing tool in town -- meet "Fanzor!" news.mit.edu/2023/fanzor-sy…

An exciting initiative funded by @NIH uses the latest genome editing tools to advance cures for #Rettsyndrome and 3 other diseases! The lead on the effort is Dr. Cathleen Lutz of @jacksonlab Learn more on our blog. reverserett.org/news/articles/…

FDA has just approved a gene therapy for hemophilia A. BioMarin’s president of R&D, Henry Fuchs, sums it up as follows: “The learning here was it doesn’t work in everybody. It doesn’t last forever. Our view is it works in enough people and long enough.” endpts.com/biomarins-hemo…

Reverse Rett Colorado is coming up on July 28. Thank you to grandparents Karen and Steve Bye for spearheading this beautiful event. Way to make a difference to the research! reverserett.org/events/reverse…