An emerging era of clinical benefit from gene therapy

Affiliations

¹ Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
² Department of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle.

Editorial

Harry L Malech et al. JAMA. 2015.

. 2015 Apr 21;313(15):1522-3.

doi: 10.1001/jama.2015.2055.

¹ Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
² Department of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle.

No abstract available

Comment on

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K, Picard C, Six E, Himoudi N, Gilmour K, McNicol AM, Hara H, Xu-Bayford J, Rivat C, Touzot F, Mavilio F, Lim A, Treluyer JM, Héritier S, Lefrère F, Magalon J, Pengue-Koyi I, Honnet G, Blanche S, Sherman EA, Male F, Berry C, Malani N, Bushman FD, Fischer A, Thrasher AJ, Galy A, Cavazzana M. Hacein-Bey Abina S, et al. JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253. JAMA. 2015. PMID: 25898053 Free PMC article. Clinical Trial.