Base Editing of Hematopoietic Stem Cells Rescues T-Cell Development for CD3d Severe Combined Immunodeficiency

McAuley, GE; Yiu, G; Newby, GA; Kang, SHL; Garibay, AJ; Butler, JA; Christian, VS; Fitz-Gibbon, S; Wong, RL; Everette, KA; Romero, Z; Wright, N; Liu, DR; Crooks, GM; Kohn, DB

MOLECULAR THERAPY, 2022; 30 (4): 231

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