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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Veröffentlicht in:Gene Ther
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2021
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Article
Online Zugang: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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