Base-Editing as a Safe and Highly Effective Alternative Treatment for X-SCID Compared to CRISPR-Cas9 Nuclease Editing with an AAV Donor
Vera Bzhilyanskaya,Julie Brault,Siyuan Liu, Nikita Kozhushko,Amanda Lawson,Mara Pavel-Dinu,Aaron B. Clark,Ronald J. Meis,Michelle Ma,Cicera R. Lazzarotto,Shengdar Q. Tsai,Xiaolin Wu,Gary Dahl,Benjamin P. Kleinstiver,Matthew H. Porteus,Harry L. Malech,Suk See De Ravin MOLECULAR THERAPY（2023）
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