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CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.
Sharma A, Boelens JJ, Cancio M, Hankins JS, Bhad P, Azizy M, Lewandowski A, Zhao X, Chitnis S, Peddinti R, Zheng Y, Kapoor N, Ciceri F, Maclachlan T, Yang Y, Liu Y, Yuan J, Naumann U, Yu VWC, Stevenson SC, De Vita S, LaBelle JL. Sharma A, et al. Among authors: zheng y. N Engl J Med. 2023 Aug 31;389(9):820-832. doi: 10.1056/NEJMoa2215643. N Engl J Med. 2023. PMID: 37646679 Clinical Trial.
Genetic therapies for the first molecular disease.
Doerfler PA, Sharma A, Porter JS, Zheng Y, Tisdale JF, Weiss MJ. Doerfler PA, et al. Among authors: zheng y. J Clin Invest. 2021 Apr 15;131(8):e146394. doi: 10.1172/JCI146394. J Clin Invest. 2021. PMID: 33855970 Free PMC article. Review.
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