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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 Buckland, Uimook Choi, Suk See De Ravin, Morna J. Dorsey, Caroline Y. Kuo, Diego León‐Rico, Christine Rivat, Natalia Izotova, Kimberly Gilmour, Katie Snell, Jinhua Xu-Bayford Dip, Jinan Darwish, Emma Morris, Dayna Terrazas, Leo D. Wang, Christopher Bauser, Tobias Paprotka, Douglas B. Kuhns, John Gregg, Hayley E. Raymond, J.K. Everett, Géraldine 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

2020Nature Medicine253 citationsDOIOpen Access PDF

Topics & Concepts

Chronic granulomatous diseaseGenetic enhancementMedicineImmunologyHaematopoiesisPrimary immunodeficiencyStem cellHematopoietic stem cell transplantationCD34Progenitor cellInternal medicineDiseaseImmune systemBiologyGeneGeneticsBiochemistryVirus-based gene therapy researchNeutrophil, Myeloperoxidase and Oxidative MechanismsCRISPR and Genetic Engineering
Lentiviral gene therapy for X-linked chronic granulomatous disease | Litcius