Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair

X-linked chronic granulomatous disease is an immunodeficiency characterized by defective production of microbicidal reactive oxygen species (ROS) by phagocytes. Causative mutations occur throughout the 13 exons and splice sites of the CYBB gene, resulting in loss of gp91(phox) protein. Here we repor...

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Publicado no:Gene Ther
Main Authors: Sweeney, Colin L., Pavel-Dinu, Mara, Choi, Uimook, Brault, Julie, Liu, Taylor, Koontz, Sherry, Li, Linhong, Theobald, Narda, Lee, Janet, Bello, Ezekiel A., Wu, Xiaolin, Meis, Ronald J., Dahl, Gary A., Porteus, Matthew H., Malech, Harry L., See De Ravin, Suk
Formato: Artigo
Idioma:Inglês
Publicado em: 2021
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8232036/
https://ncbi.nlm.nih.gov/pubmed/33712802
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-021-00251-z
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