Fetal Hemoglobin

Sustained Genetic Silencing of BCL11A Using shmiR Vector in Sickle Cell Disease: Long-Term Safety and Efficacy

This article reviews the first-in-human study demonstrating durable and safe posttranscriptional silencing of BCL11A in sickle cell disease patients using a shmiR vector, resulting in stable fetal hemoglobin induction and clinical benefit o