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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Gene Ther
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2021
Gaiak:
Article
Sarrera elektronikoa: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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