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Gene Therapy Treats Sickle Cell Disease in Teenager Using Lentiviral Vector.

Gene therapy that delivered an antisickling variant of hemoglobin in an autologous hematopoietic stem cell (HSC) transplant has ameliorated symptoms of severe sickle cell disease (SCD) in a 15-year-old boy, according to a report published in the March 2 issue of the New England Journal of Medicine.

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