Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.

ABSTRACT: Curative therapies for sickle cell disease (SCD) include allogeneic human leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell transplant (HCT), gene therapy, and gene editing. However, comparative trial data that might facilitate selecting one curative therapy over another are unavailable. New strategies to decrease graft rejection and graft-versus-host disease (GVHD) risks are needed to expand haploidentical HCT. Myeloablative gene therapy and gene editing also has limitations. Herein, we review recent studies on curative therapies for SCD in the past 5 years.

SUBMITTER: Lawal RA 

PROVIDER: S-EPMC9681017 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature