[gavinray]

"Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease"

https://www.nejm.org/doi/full/10.1056/NEJMoa2504747

[gwern]

Note: from May 2025. https://gwern.net/doc/genetics/editing/2025-musunuru.pdf

[tomhow]

Thanks! We've put this link in the header of the thread.