Gene Therapy: A New Hope in Sickle Cell Disease Treatment

Abstract

Sickle cell disease (SCD) is a lifelong disease requiring expensive treatment for management and limited curative options until the last few years. Gene therapy has emerged as a curative option for SCD, with two approved therapies available to SCD patients aged ≥ 12 years. Consideration must be considered regarding the ethics, efficacy, management requirements, education, and counseling needs of patients and their parents. Current and future practices will need to advocate for improved access and affordability of this specialized care and address the unknown and less defined areas of gene therapy pertaining to SCD through research.