Gene therapy for sickle cell disease.

Abstract

Sickle cell disease (SCD) is potentially curable after allogeneic hematopoietic stem cell transplantation (HSCT) or autologous HSCT after ex vivo genetic modification. Autologous HSCT with gene therapy has the potential to overcome many of the limitations of allogeneic HSCT that include the lack of suitable donors, graft-versus-host disease, the need for immune suppression, and the potential for graft rejection. Significant progress in gene therapy for SCD has been made over the past several decades, now with a growing number of clinical trials investigating various gene addition and gene editing strategies. Available results from a small number of patients, some with relatively short follow-up, are promising as a potentially curative strategy, with current efforts focused on continuing to improve the efficacy, durability, and safety of gene therapies for the cure of SCD.