Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
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Published:2021-05-27
Issue:21
Volume:384
Page:2002-2013
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ISSN:0028-4793
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Container-title:New England Journal of Medicine
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language:en
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Short-container-title:N Engl J Med
Author:
Kohn Donald B.1, Booth Claire1, Shaw Kit L.1, Xu-Bayford Jinhua1, Garabedian Elizabeth1, Trevisan Valentina1, Carbonaro-Sarracino Denise A.1, Soni Kajal1, Terrazas Dayna1, Snell Katie1, Ikeda Alan1, Leon-Rico Diego1, Moore Theodore B.1, Buckland Karen F.1, Shah Ami J.1, Gilmour Kimberly C.1, De Oliveira Satiro1, Rivat Christine1, Crooks Gay M.1, Izotova Natalia1, Tse John1, Adams Stuart1, Shupien Sally1, Ricketts Hilory1, Davila Alejandra1, Uzowuru Chilenwa1, Icreverzi Amalia1, Barman Provaboti1, Campo Fernandez Beatriz1, Hollis Roger P.1, Coronel Maritess1, Yu Allen1, Chun Krista M.1, Casas Christian E.1, Zhang Ruixue1, Arduini Serena1, Lynn Frances1, Kudari Mahesh1, Spezzi Andrea1, Zahn Marco1, Heimke Rene1, Labik Ivan1, Parrott Roberta1, Buckley Rebecca H.1, Reeves Lilith1, Cornetta Kenneth1, Sokolic Robert1, Hershfield Michael1, Schmidt Manfred1, Candotti Fabio1ORCID, Malech Harry L.1, Thrasher Adrian J.1, Gaspar H. Bobby1
Affiliation:
1. From the Departments of Microbiology, Immunology, and Molecular Genetics (D.B.K., K.L.S., D.A.C.-S., D.T., A.D., A. Icreverzi, P.B., B.C.F., R.P.H., M.C., A.Y., K.M.C., C.E.C., R.Z.), Pediatrics (D.B.K., T.B.M., S.D.O., S.S.), and Pathology and Laboratory Medicine (G.M.C.) and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research (D.B.K., G.M.C.), University of California, Los Angeles (UCLA), and the Department of Pharmaceutical Services, Ronald Reagan UCLA Medical Center (J.T.)...
Funder
National Gene Vector Biorepository California Institute for Regenerative Medicine Medical Research Council National Institute for Health Research Biomedical Research Centre at the Great Ormond Street Hospital for Children NHS Foundation Trust and University College London Orchard Therapeutics Ltd National Institutes of Health National Heart, Lung, and Blood Institute National Human Genome Research Institute National Institute of Allergy and Infectious Diseases
Publisher
Massachusetts Medical Society
Cited by
112 articles.
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