September 10, 2019, Vol 322, No. 10, Pages 903-1022
Scientific Discovery and the Future of Medicine
Accelerating the Science of SCD Therapies—Is a Cure Possible?
Edward J. Benz Jr, MD; Traci Heath Mondoro, PhD; Gary H. Gibbons, MD
JAMA. 2019;322(10):921-922. doi:10.1001/jama.2019.11419
This Viewpoint reviews the molecular basis of sickle cell disease (SCD), the history of treatments for the disease, and recent progress toward curative therapies including HSCT and gene therapy and editing, emphasizing that research and innovation needs to take into account patient priorities and perspectives given the history of research mistreatment of minority groups most affected by SCD.