Sickle cell disease is a group of inherited blood disorders characterized by chronic anemia, episodes of pain, and other serious complications, including stroke. Researchers, including those at Children’s Hospital of Philadelphia, have been investigating the use of gene therapy to treat the condition, giving those living with sickle cell disease much hope. In this short documentary by the New England Journal of Medicine, Dr. Alexis Thompson, our Chief of Hematology, discusses the early successes of gene therapy, as well as its risks and benefits.
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Sickle cell disease is a group of inherited blood disorders characterized by chronic anemia, episodes of pain, and other serious complications, including stroke. Researchers, including those at Children’s Hospital of Philadelphia, have been investigating the use of gene therapy to treat the condition, giving those living with sickle cell disease much hope. In this short documentary by the New England Journal of Medicine, Dr. Alexis Thompson, our Chief of Hematology, discusses the early successes of gene therapy, as well as its risks and benefits.
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