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Genetic Treatments for Sickle Cell
For decades physicians have known that a few children like Ceniya have unusual genetic mutations that counteract the effects of the sickle-cell flaw. Researchers would like to re-create their uncommon physiology in everyone with sickle-cell anemia. Though not technically a cure, the compensatory treatment would spare many of the 300,000 infants around the world who are born every year with sickle cell and who often do not live beyond childhood. It would also make life a lot easier for the more than 70,000 individuals living with the disease in the U.S., who, despite treatment that mitigates some of the most serious effects of the condition, often die in their 40s.
Investigators are now beginning to test such approaches, which depend on the precise alteration, or editing, of certain genes using new techniques in genetic engineering. (As will be addressed shortly, providing the compensatory mechanism should be easier to achieve than fixing the original sickle-cell genetic defect.)
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.