Hematopoietic Stem-Cell Gene Therapy for Cystinosis.

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Bibliographic Details
Title: Hematopoietic Stem-Cell Gene Therapy for Cystinosis.
Authors: Barshop BA; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla., Ball ED; Department of Medicine, Division of Blood and Marrow Transplantation, University of California, San Diego, La Jolla., Benador N; Department of Pediatrics, Division of Nephrology, University of California, San Diego, La Jolla.; Rady Children's Hospital, San Diego, CA., Trauner D; Department of Neurosciences, University of California, San Diego, La Jolla., Phillips S; Rady Children's Hospital, San Diego, CA.; Department of Pediatrics, Division of Endocrinology, University of California, San Diego, La Jolla., Dohil R; Rady Children's Hospital, San Diego, CA.; Department of Pediatrics, Division of Gastroenterology, University of California, San Diego, La Jolla., Afshari NA; Department of Ophthalmology, University of California, San Diego, La Jolla., Roy S; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles., Campo Fernandes B; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles., Kohn D; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles., Shayan K; Rady Children's Hospital, San Diego, CA.; Department of Pathology, University of California, San Diego, La Jolla.; Department of Pediatrics, University of California, San Diego, La Jolla., Everett JK; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia., Bushman FD; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia., Midgley J; Department of Pediatrics, University of Calgary, Calgary, AB, Canada., Liang H; Department of Ophthalmology III, Quinze-Vingts National Ophthalmology Hospital, DHU Sight Restore, INSERM-DHOS CIC, Paris., Sawyers A; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla., Gangoiti JA; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla., Panchal M; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla., Ahmed I; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla., Cherqui S; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.
Source: The New England journal of medicine [N Engl J Med] 2026 Feb 19; Vol. 394 (8), pp. 753-762.
Publication Type: Journal Article; Clinical Trial, Phase I; Clinical Trial, Phase II
Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1533-4406
DOI:10.1056/NEJMoa2506431