Article
CRISPR/Cas9-mediated Cxcr4 disease allele inactivation for gene therapy in a mouse model of WHIM syndrome.
Blood - 6 Jul 2023
Gao Ji-Liang, Owusu-Ansah Albert, Yang Alexander, Yim Erin, McDermott David H, Jacobs Paejonette, Majumdar Shamik, Choi Uimook, Sweeney Colin L, Malech Harry L, Murphy Philip M
Abstract excerpt
WHIM syndrome is an autosomal dominant immunodeficiency disorder caused by gain-of-function mutations in chemokine receptor CXCR4 that promote severe panleukopenia because of retention of mature leukocytes in the bone marrow (BM). We previously reported that Cxcr4-haploinsufficient (Cxcr4+/o) hematopoietic stem cells (HSCs) have a strong selective advantage for durable hematopoietic reconstitution over wild-type...
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