Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1-Reference-Cited by-同舟云学术

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1

Published:2006-04 Issue:4 Volume:12 Page:401-409
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

Ott Marion G,Schmidt Manfred,Schwarzwaelder Kerstin,Stein Stefan,Siler Ulrich,Koehl Ulrike,Glimm Hanno,Kühlcke Klaus,Schilz Andrea,Kunkel Hana,Naundorf Sonja,Brinkmann Andrea,Deichmann Annette,Fischer Marlene,Ball Claudia,Pilz Ingo,Dunbar Cynthia,Du Yang,Jenkins Nancy A,Copeland Neal G,Lüthi Ursula,Hassan Moustapha,Thrasher Adrian J,Hoelzer Dieter,von Kalle Christof,Seger Reinhard,Grez Manuel

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Link

http://www.nature.com/articles/nm1393.pdf

Reference47 articles.

1. Cavazzana-Calvo, M. et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669–672 (2000).

2. Aiuti, A. et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296, 2410–2413 (2002).

3. Hacein-Bey-Abina, S. et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N. Engl. J. Med. 346, 1185–1193 (2002).

4. Gaspar, H.B. et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364, 2181–2187 (2004).

5. Malech, H.L. et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc. Natl. Acad. Sci. USA 94, 12133–12138 (1997).

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