Mitigation of cerebellar neuropathy in globoid cell leukodystrophy mice by AAV-mediated gene therapy》.pdfVIP

Gene 571 (2015) 81–90 Contents lists available at ScienceDirect Gene journal homepage: www. /locate/gene Research paper Mitigation of cerebellar neuropathy in globoid cell leukodystrophy mice by AAV-mediated gene therapy a,b,c,⁎ h i a c Dar-Shong Lin , Chung-Der Hsiao , Allan Yueh-Luen Lee , Che-Sheng Ho , Hsuan-Liang Liu , Tuen-Jen Wang f, Yuan-Ren Jian g, Jui-Cheng Hsu g, Zon-Darr Huang g, Tsung-Han Lee g, Ming-Fu Chiang d,e,⁎ a Department of Pediatrics, Mackay Memorial Hospital, Taipei, Taiwan b Department of Medicine, Mackay Medical College, New Taipei, Taiwan c Department of Chemical Engineering and Biotechnology, National Taipei University of Technology, Taipei, Taiwan d Department of Neurosurgery, Mackay Memorial Hospital, Taipei, Taiwan e Institute of Injury Prevention and Control, Taipei Medical University, Taipei, Taiwan f Department of Laboratory Medicine, Mackay Memorial Hospital, Taipei, Taiwan g Department of Medical Research, Mackay Memorial Hospital, Taipei, Taiwan h Department of Bioscience Technology, Chung Yuan Christian University, Chung-Li, Taiwan i National Institute of Cancer Research, National Health Research Institutes, Miaoli, Taiwan a r t i c l e i n f o a b s t r a c t Article history: Globoid cell leukodystrophy (GLD) is an autosomal recessive, lysosomal storage disease caused by deficiency of Receiv