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Gene-Edited ‘Supercells’ Make Progress In Fight Against Sickle Cell Disease
Doctors are reporting the first evidence that genetically edited cells could offer a safe way to treat sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world.
Billions of cells that were genetically modified with the powerful gene-editing technique called CRISPR have started working, as doctors had hoped, inside the body of the first sickle cell patient to receive the experimental treatment, according to highly anticipated data released Tuesday.
The edited cells are producing a crucial protein at levels that have already exceeded what doctors thought would be needed to alleviate the excruciating, life-threatening complications of the genetic blood disorder, the early data show. Moreover, the cells appear to have already started to spare the patient from the agonizing attacks of pain that are the hallmark of the disorder.
“We are very, very excited,” says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating the patient. “This preliminary data shows for the first time that gene editing has actually helped a patient with sickle cell disease. This is definitely a huge deal.”
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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.