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Gene therapy may help cure sickle cell disease, study says
A gene therapy that could provide a permanent cure for sickle cell disease continues to show success through a third wave of patients, researchers report.
The therapy, LentiGlobin, restored normal blood function in 35 sickle cell patients who had the one-time procedure, according to clinical trial findings published Sunday in the New England Journal of Medicine.
The patients are all now producing stable amounts of normal red blood cells containing healthy hemoglobin, said lead researcher Dr. Julie Kanter, director of the University of Alabama at Birmingham’s Adult Sickle Cell Clinic.
In addition, they have not suffered the severe pain episodes that come with sickle cell disease, she said.
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