Article
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.
Science translational medicine - 16 Oct 2024
Bzhilyanskaya Vera, Ma Linyuan, Liu Siyuan, Fox Lauren R, Whittaker Madelynn N, Meis Ronald J, Choi Uimook, Lawson Amanda, Ma Michelle, Theobald Narda, Burkett Sandra, Sweeney Colin L, Lazzarotto Cicera R, Tsai Shengdar Q, Lack Justin B, Wu Xiaolin, Dahl Gary A, Malech Harry L, Kleinstiver Benjamin P, De Ravin Suk See
Abstract excerpt
X-linked chronic granulomatous disease (X-CGD) is an inborn error of immunity (IEI) resulting from genetic mutations in the cytochrome b-245 beta chain (CYBB) gene. The applicability of base editors (BEs) to correct mutations that cause X-CGD is constrained by the requirement of Cas enzymes to recognize specific protospacer adjacent motifs (PAMs). Our recently engineered PAMless Cas enzyme, SpRY, can overcome the...
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