Post-Transcriptional Genetic Silencing of <i>BCL11A</i> to Treat Sickle Cell Disease

Abstract

This study validates BCL11A inhibition as an effective target for HbF induction and provides preliminary evidence that shmiR-based gene knockdown offers a favorable risk-benefit profile in sickle cell disease. (Funded by the National Institutes of Health; ClinicalTrials.gov number, NCT03282656).