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...

全面介紹

Na minha lista:
書目詳細資料
發表在: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
格式: Artigo
語言:Inglês
出版: 2021
主題:
Article
在線閱讀: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
標簽: 添加標簽
沒有標簽, 成為第一個標記此記錄!

相似書籍