More than 50 years after the cause of sickle-cell disease was discovered, a dozen treatments for the painful and life-shortening inherited condition offer hope for long-overlooked patients.
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Now it’s getting the attention of cutting-edge researchers. Bluebird Bio Inc. hopes to cure patients with a single injection of its gene therapy one day. Farther down the road are even more potent and permanent experiments using the gene-editing technology Crispr, an approach that hasn’t been done in humans yet.
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Bluebird’s LentiGlobin gene therapy and the promise of Crispr were in the limelight at the American Society of Hematology, or ASH, meeting in Atlanta this weekend, capturing the imagination of what could done to cure diseases caused by defective genes. Coming sooner will be more conventional therapies to treat symptoms, like Global Blood Therapeutics Inc.’s voxelotor, one of the first drugs designed specifically for sickle-cell disease.
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“The curative options are exciting, but they won’t be available to a majority of patients for a long time,” said Carolyn Hoppe, associate hematologist from University of California San Francisco’s Benioff Children’s Hospital in Oakland, and lead researcher on the voxelotor trial. “People are focusing on getting a cocktail of drugs that can be given safely that work differently until we can get to the point of cure.”
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