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Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.

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第一作者： Heather L,Gray-Edwards

第一单位： 1 Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama.

作者： Heather L,Gray-Edwards ^[1] ; Ashley N,Randle ^[1] ; Stacy A,Maitland ^[2] ; Hector R,Benatti ^[1] ; Spencer M,Hubbard ^[1] ; Peter F,Canning ^[1] ; Matthew B,Vogel ^[1] ; Brandon L,Brunson ^[3] ; Misako,Hwang ^[1] ; Lauren E,Ellis ^[1] ; Allison M,Bradbury ^[4] ; Atoska S,Gentry ^[1] ; Amanda R,Taylor ^[5] ; Anne A,Wooldridge ^[5] ; Dewey R,Wilhite ^[3] ; Randolph L,Winter ^[5] ; Brian K,Whitlock ^[6] ; Jacob A,Johnson ^[5] ; Merilee,Holland ^[5] ; Nouha,Salibi ^[7] ; Ronald J,Beyers ^[8] ; James L,Sartin ^[3] ; Thomas S,Denney ^[8] ; Nancy R,Cox ^[9] ; Miguel,Sena-Esteves ^[2] ; Douglas R,Martin ^[4]

作者单位： 1 Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama. ^[1] 2 Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts. ^[2] 3 Department of Anatomy, Physiology, and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama. ^[3] 1 Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama.;3 Department of Anatomy, Physiology, and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama. ^[4] 4 Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, Auburn, Alabama. ^[5] 5 Department of Large Animal Clinical Sciences, College of Veterinary Medicine, University of Tennessee , Knoxville, Tennessee. ^[6] 6 MR R&D Siemens Healthcare, Malvern, Pennsylvania. ^[7] 7 Department of Electrical and Computer Engineering, Auburn University, Auburn, Alabama. ^[8] 1 Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama.;8 Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, Alabama. ^[9]

关键词 Tay-Sachs adeno-associated virus animal model gangliosidosis gene therapy sheep