Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency-Reference-Cited by-同舟云学术

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Published:2021-05-27 Issue:21 Volume:384 Page:2002-2013
ISSN:0028-4793
Container-title:New England Journal of Medicine
language:en
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 Fabio¹^ORCID,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

Subject

General Medicine

Link

http://www.nejm.org/doi/pdf/10.1056/NEJMoa2027675

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