Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease.

Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, et al. Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.