exa-cel
Vertex pharmaceuticals,CRISPR therapeutics AG
[]
not every day you see 3-year follow-up of one-shot genome editing to keep 31/31 people with severe sickle-cell disease without any crises and 42/44 with beta-thalassemia without transfusions #eha2022 
physician-scientist, author, editor  
https://t.co/vwstu2BYrC