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Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.

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第一作者： Allison M,Bradbury

第一单位： Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

作者： Allison M,Bradbury ^[1] ; Jessica H,Bagel ^[1] ; Duc,Nguyen ^[2] ; Erik A,Lykken ^[3] ; Jill,Pesayco Salvador ^[4] ; Xuntian,Jiang ^[5] ; Gary P,Swain ^[1] ; Charles A,Assenmacher ^[6] ; Ian J,Hendricks ^[1] ; Keiko,Miyadera ^[1] ; Rebecka S,Hess ^[1] ; Arielle,Ostrager ^[1] ; Patricia,ODonnell ^[1] ; Mark S,Sands ^[5] ; Daniel S,Ory ^[5] ; G Diane,Shelton ^[4] ; Ernesto R,Bongarzone ^[2] ; Steven J,Gray ^[3] ; Charles H,Vite ^[1]

作者单位： Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA. ^[1] Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, Illinois, USA. ^[2] Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA. ^[3] Department of Pathology, School of Medicine, University of California San Diego, La Jolla, California, USA. ^[4] Department of Medicine, Washington University School of Medicine, St. Louis, Missouri, USA. ^[5] Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA. ^[6]

关键词 Demyelinating disorders Gene therapy Neurological disorders Neuroscience Therapeutics

医学主题词动物(Animals);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);狗(Dogs);半乳糖神经酰胺酶(Galactosylceramidase);脑白质营养不良, 球样细胞(Leukodystrophy, Globoid Cell)

DOI 10.1172/JCI133953

PMID 32773406

发布时间 2021-11-11

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准确一般字义偏差语意偏离译文作用小提交