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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.NEXT ARTICLE
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...