Gene therapy offers hope for treatment of sickle cell anemia

October 01, 2016


Scientists are finalizing plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anemia, the Guardian reports. The technique could begin trials next year, say researchers.

“We have known exactly what is the cause of sickle cell anemia for 60 years, but it has been enormously difficult to turn that information into a treatment,” Stuart Orkin, MD, tells the Guardian. “There are a million steps between the lab bench and the clinic, it turns out. However, I think we are closer.”

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