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Hematopoietic Stem-Cell Gene Therapy for Cystinosis.

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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
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  Data: Hematopoietic Stem-Cell Gene Therapy for Cystinosis.
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  Data: <searchLink fieldCode="AU" term="%22Barshop+BA%22">Barshop BA</searchLink>; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Ball+ED%22">Ball ED</searchLink>; Department of Medicine, Division of Blood and Marrow Transplantation, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Benador+N%22">Benador N</searchLink>; Department of Pediatrics, Division of Nephrology, University of California, San Diego, La Jolla.; Rady Children's Hospital, San Diego, CA.<br /><searchLink fieldCode="AU" term="%22Trauner+D%22">Trauner D</searchLink>; Department of Neurosciences, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Phillips+S%22">Phillips S</searchLink>; Rady Children's Hospital, San Diego, CA.; Department of Pediatrics, Division of Endocrinology, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Dohil+R%22">Dohil R</searchLink>; Rady Children's Hospital, San Diego, CA.; Department of Pediatrics, Division of Gastroenterology, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Afshari+NA%22">Afshari NA</searchLink>; Department of Ophthalmology, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Roy+S%22">Roy S</searchLink>; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Campo+Fernandes+B%22">Campo Fernandes B</searchLink>; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Kohn+D%22">Kohn D</searchLink>; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Shayan+K%22">Shayan K</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Everett+JK%22">Everett JK</searchLink>; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Bushman+FD%22">Bushman FD</searchLink>; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Midgley+J%22">Midgley J</searchLink>; Department of Pediatrics, University of Calgary, Calgary, AB, Canada.<br /><searchLink fieldCode="AU" term="%22Liang+H%22">Liang H</searchLink>; Department of Ophthalmology III, Quinze-Vingts National Ophthalmology Hospital, DHU Sight Restore, INSERM-DHOS CIC, Paris.<br /><searchLink fieldCode="AU" term="%22Sawyers+A%22">Sawyers A</searchLink>; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Gangoiti+JA%22">Gangoiti JA</searchLink>; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Panchal+M%22">Panchal M</searchLink>; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Ahmed+I%22">Ahmed I</searchLink>; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.<br /><searchLink fieldCode="AU" term="%22Cherqui+S%22">Cherqui S</searchLink>; Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.
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  Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2026 Feb 19; Vol. 394 (8), pp. 753-762.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Massachusetts+Medical+Society%22">Massachusetts Medical Society </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>0255562 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1533-4406 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200284793%22">00284793 </searchLink><i>NLM ISO Abbreviation: </i>N Engl J Med <i>Subsets: </i>MEDLINE
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