Here's a 🧵 summing up our latest preprint. 👇👇biorxiv.org/content/10.110…

Remarkable speech. Addressed to the Russian people, it should also be heard everywhere.

A new one-time base-editing treatment for spinal muscular atrophy — a neuromuscular disease that is the leading genetic cause of infant mortality — restored motor function and extended lifespan in a mouse model. broad.io/SMAbaseediting

A research team led by #geneediting pioneer David R. Liu has published how it has applied the technology to develop a one-time treatment for #spinalmuscularatrophy that showed effectiveness in cell and mouse models. Learn more: ow.ly/37o950NwnsN

Approved genomic therapies for SMA have, to date, saved 14,000 children from certain death. This work is a key step towards a one-time treatment that, one hopes, will be more accessible on a global scale.

Very excited to announce the publication of our corrective prime editing strategy for sickle cell disease! It was an honor to lead this work with David’s support and in collaboration with colleagues from St. Jude and the NIH.

Check out this beautiful functional genomics study combining genome-wide CRISPR screening, human Biobank data and gene network analysis to identify new therapeutic candidate genes for heart disease that increase LDL uptake and lower cholesterol in mice! Great work all! 🧬🔬🐁

I was taken by surprise this morning when congratulatory emails started arriving in my inbox. I'm incredibly honored, grateful, and humbled to be included among such an esteemed group of scientists, many of whom I’ve admired for decades. While this recognition is individual,

🙏🙏 to all speakers, presenters, & attendees for making this conference a success! 3 intense days of impressive science. 🧬🔬 Grateful to work with these co-orgs! 🙏 Kristin Knouse AndrewAnzalone Morgan Maeder Mandana Arbab Britt Adamson Jonathan Strecker MattStanton JeffQuinn

Thank you Matt and World Muscle Society for the honor! 😊 Żaneta Matuszek David R. Liu

SCGE Phase 2 researcher Mandana Arbab (Mandana Arbab) is presenting on precision genome editing for treating genetic neurological diseases. Check it out this morning at #ASGCT2024