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...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:Nat Med
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2020
Témata:
Article
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky