Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome
Copyright policy )Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome
Key PointsThe CD40LG locus can be specifically targeted and repaired in primary human T cells by insertion of a spliced CD40LG complementary DNA. Gene editing restores regulated CD40L expression in X-HIGM T cells, reconstituting B-cell immunoglobulin class switching.
- University of Mary United States
- University of Washington United States
- Seattle Children's Research Institute United States
Gene Editing, Male, B-Lymphocytes, Hyper-IgM Immunodeficiency Syndrome, Type 1, T-Lymphocytes, CD40 Ligand, Mice, SCID, Immunoglobulin Class Switching, Mice, Enhancer Elements, Genetic, Gene Expression Regulation, Mice, Inbred NOD, Animals, Humans, Female, Somatic Hypermutation, Immunoglobulin, 3' Untranslated Regions, Targeted Gene Repair
Gene Editing, Male, B-Lymphocytes, Hyper-IgM Immunodeficiency Syndrome, Type 1, T-Lymphocytes, CD40 Ligand, Mice, SCID, Immunoglobulin Class Switching, Mice, Enhancer Elements, Genetic, Gene Expression Regulation, Mice, Inbred NOD, Animals, Humans, Female, Somatic Hypermutation, Immunoglobulin, 3' Untranslated Regions, Targeted Gene Repair
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