Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

ABSTRACT: The pre-clinical efficacy and safety data support the initiation of a phase I/II clinical trial for β-globin gene correction in patient-derived hematopoietic stem cells for the treatment of sickle cell disease.

ORGANISM(S): Homo sapiens

PROVIDER: GSE163912 | GEO | 2021/05/16

REPOSITORIES: GEO