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Experimental Gene Therapy Reverses Sickle Cell Disease for Years
A study of an investigational gene therapy for sickle cell disease has found that a single dose restored blood cells to their normal shape and eliminated the most serious complication of the disease for at least three years in some patients.
Four patients at Columbia University Irving Medical Center/NewYork-Presbyterian participated in the multicenter study, the first to report on such long-term outcomes of a sickle cell gene therapy. The study was published online Dec. 12 in the New England Journal of Medicine with John F. Tisdale, MD, senior investigator at the NIH’s National Heart, Lung and Blood Institute, as corresponding author.
The single-dose therapy, tested on 35 adults and adolescents with sickle cell disease, essentially corrected the shape of the patient’s red blood cells, but also completely eliminated episodes of severe pain, caused when rigid, crescent-shaped red blood cells clump together and block blood vessels. The painful episodes often result in widespread organ damage. Such episodes are a frequent cause of emergency department visits and hospitalizations and lead to early death.
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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.