CRISPR-Cas9 Gene Therapy Cure for Sickle Cell Disease
TL;DR Summary: A comprehensive look at the clinical validation of exagamglogene autotemcel, the first CRISPR-based gene editing therapy approved for sickle cell disease.
Sickle cell disease, a debilitating inherited blood disorder, has met its match with the historic approval and successful implementation of CRISPR-based gene editing.
Mechanism of Action
The treatment, known as Casgevy, targets the BCL11A gene in autologous hematopoietic stem cells to reactivate fetal hemoglobin production, effectively preventing the sickling of red blood cells.
Discussion & Comments