Lentiviral gene therapy for X-linked chronic granulomatous disease

Donald B. Kohn, Claire Booth, Elizabeth M. Kang, Sung-Yun Pai, Kit L. Shaw, Giorgia Santilli, Myriam Armant, Karen F. Buckland, Uimook Choi, Suk See De Ravin, Morna J. Dorsey, Caroline Y. Kuo, Diego Leon-Rico, Christine Rivat, Natalia Izotova, Kimberly Gilmour, Katie Snell, Jinhua Xu-Bayford Dip, Jinan Darwish, Emma C. Morris, Dayna Terrazas, Leo D. Wang, Christopher A. Bauser, Tobias Paprotka, Douglas B. Kuhns, John Gregg, Hayley E. Raymond, John K. Everett, Geraldine Honnet, Luca Biasco, Peter E. Newburger, Frederic D. Bushman, Manuel Grez, H. Bobby Gaspar, David A. Williams, Harry L. Malech, Anne Galy, Adrian J. Thrasher & the Net4CGD consortium

Monday, January 27, 2020
Published in Nature Medicine
https://www.nature.com/articles/s41591-019-0735-5
before and after gene therapy cell comparison image
At left, image shows white blood cells (red) from one of the X-CGD clinical trial participants before gene therapy. At right, after gene therapy, white blood cells from the same patient show the presence of the chemicals (blue) needed to attack and destroy bacteria and fungus.