Sickle cell disease (SCD) is the most common inherited red blood cell disorder in the United States, affecting 70 000 to 100 000 Americans and causing a range of serious medical complications. Although the cause of SCD was established decades ago, existing therapies have varied effectiveness and side effects, and development of novel therapies has been slow. The limitations of existing treatment options highlight the need for new therapies that are aligned with the desires of the community.
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