ARTICLE | Distillery Therapeutics
Therapeutics: Hemoglobin subunit γ 1 (HBG1); HBG2; CRISPR-associated protein 9 (Cas9)
September 22, 2016 7:00 AM UTC
Patient sample and cell culture studies suggest enhancing fetal hemoglobin production in hematopoietic stem and progenitor cells (HSPCs) using CRISPR-based gene editing could help treat SCD. In HSPCs ...