CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

XMEN disease, defined as “X-linked MAGT1 deficiency with increased susceptibility to Epstein-Barr virus infection and N-linked glycosylation defect,” is a recently described primary immunodeficiency marked by defective T cells and natural killer (NK) cells. Unfortunately, a potentially curative hema...

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Veröffentlicht in:	Blood 2021-12, Vol.138 (26), p.2768-2780
Hauptverfasser:	Brault, Julie, Liu, Taylor, Bello, Ezekiel, Liu, Siyuan, Sweeney, Colin L., Meis, Ronald J., Koontz, Sherry, Corsino, Cristina, Choi, Uimook, Vayssiere, Guillaume, Bosticardo, Marita, Dowdell, Kennichi, Lazzarotto, Cicera R., Clark, Aaron B., Notarangelo, Luigi D., Ravell, Juan C., Lenardo, Michael J., Kleinstiver, Benjamin P., Tsai, Shengdar Q., Wu, Xiaolin, Dahl, Gary A., Malech, Harry L., De Ravin, Suk See
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Cation Transport Proteins - deficiency Cation Transport Proteins - genetics Cells, Cultured CRISPR-Cas Systems Female Gene Editing - methods Gene Therapy Genetic Therapy Hematopoietic Stem Cell Transplantation Hematopoietic Stem Cells - metabolism Hematopoietic Stem Cells - pathology Humans Lymphocytes - metabolism Lymphocytes - pathology Male Mice, Inbred NOD X-Linked Combined Immunodeficiency Diseases - genetics X-Linked Combined Immunodeficiency Diseases - pathology X-Linked Combined Immunodeficiency Diseases - therapy
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