Lentiviral gene therapy for X-linked chronic granulomatous disease

Chronic granulomatous disease (CGD) is a rare inherited disorder of phagocytic cells(1,2). We report initial results of nine severely affected X-linked CGD (X-CGD) patients who received ex vivo autologous CD34+ hematopoietic stem and progenitor cell-based lentiviral gene therapy following myeloablat...

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Bibliografske podrobnosti
izdano v:Nat Med
Main Authors: Kohn, Donald B., Booth, Claire, Kang, Elizabeth M., Pai, Sung-Yun, Shaw, Kit L., Santilli, Giorgia, Armant, Myriam, Buckland, Karen F., Choi, Uimook, De Ravin, Suk See, Dorsey, Morna J., Kuo, Caroline Y., Leon-Rico, Diego, Rivat, Christine, Izotova, Natalia, Gilmour, Kimberly, Snell, Katie, Dip, Jinhua Xu-Bayford, Darwish, Jinan, Morris, Emma C., Terrazas, Dayna RN, Wang, Leo D., Bauser, Christopher A., Paprotka, Tobias, Kuhns, Douglas B., Gregg, John, Raymond, Hayley E., Everett, John K., Honnet, Geraldine, Biasco, Luca, Newburger, Peter E., Bushman, Frederic D., Grez, Manuel, Gaspar, H. Bobby, Williams, David A., Malech, Harry L., Galy, Anne, Thrasher, Adrian J.
Format: Artigo
Jezik:Inglês
Izdano: 2020
Teme:
Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC7115833/
https://ncbi.nlm.nih.gov/pubmed/31988463
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41591-019-0735-5
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