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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8232036/
https://www.ncbi.nlm.nih.gov/pubmed/33712802
http://dx.doi.org/10.1038/s41434-021-00251-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8232036/
https://www.ncbi.nlm.nih.gov/pubmed/33712802
http://dx.doi.org/10.1038/s41434-021-00251-z

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