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Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model.

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第一作者： Heather L,Gray-Edwards

第一单位： Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA.

作者： Heather L,Gray-Edwards ^[1] ; Debra S,Regier ^[2] ; Jamie L,Shirley ^[1] ; Ashley N,Randle ^[1] ; Nouha,Salibi ^[3] ; Sarah E,Thomas ^[2] ; Yvonne L,Latour ^[2] ; Jean,Johnston ^[2] ; Gretchen,Golas ^[2] ; Annie S,Maguire ^[1] ; Amanda R,Taylor ^[4] ; Donald C,Sorjonen ^[4] ; Victoria J,McCurdy ^[5] ; Peter W,Christopherson ^[6] ; Allison M,Bradbury ^[5] ; Ronald J,Beyers ^[7] ; Aime K,Johnson ^[4] ; Brandon L,Brunson ^[8] ; Nancy R,Cox ^[9] ; Henry J,Baker ^[1] ; Thomas S,Denney ^[7] ; Miguel,Sena-Esteves ^[10] ; Cynthia J,Tifft ^[2] ; Douglas R,Martin ^[11]

作者单位： Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA. ^[1] National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA. ^[2] MR R&D, Siemens Healthcare, Malvern, PA 19355, USA. ^[3] Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA. ^[4] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA; Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA. ^[5] Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA. ^[6] Department of Electrical and Computer Engineering, Auburn University, Auburn, AL 36849, USA. ^[7] Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA. ^[8] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA; Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA. ^[9] Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01655, USA. ^[10] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA; Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA. Electronic address: martidr@auburn.edu. ^[11]

关键词 AAV gene therapy gangliosidosis lysosomal storage disorders neurodegeneration