Exonic Knockout and Knockin Gene Editing in Hematopoietic Stem and Progenitor Cells Rescues RAG1 Immunodeficiency

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Investigators developed gene editing strategies targeting a corrective sequence into the human RAG1 gene by homology-directed repair and validated them by tailored 2D, 3D, and in vivo xenotransplant platforms to assess rescue of expression and function.
[Science Translational Medicine]
Abstract