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Highly efficient therapeutic gene editing of human hematopoietic stem cells

2 monthes ago   |   By Harvard

Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, and Bauer DE. 2019. “ Highly efficient therapeutic gene editing of human hematopoietic stem cells .” Nature Medicine, 25, 5, Pp. 776-783. Abstract Re-expression of the paralogous γ-globin genes could be a universal strategy to ameliorate the severe β-globin disorders sickle cell disease and β-thalassemia by induction of fetal hemoglobin 1. Previously, we and others...
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