CRISPR–Cas9-Mediated Gene Editing of the BCL11A Enhancer for Pediatric β00 Transfusion-Dependent β-Thalassemia

The authors report the preliminary results of an ongoing phase I/II trial evaluating safety and efficacy of gene editing therapy in children with blood transfusion-dependent β-thalassemia.
[Nature Medicine]

Stay up-to-date with your field!

Subscribe for free weekly science newsletters.

Related News