Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay–Sachs Disease-Reference-Cited by-同舟云学术

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

Published:2018-03 Issue:3 Volume:29 Page:312-326
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Gray-Edwards Heather L.¹,Randle Ashley N.¹,Maitland Stacy A.²,Benatti Hector R.¹,Hubbard Spencer M.¹,Canning Peter F.¹,Vogel Matthew B.¹,Brunson Brandon L.³,Hwang Misako¹,Ellis Lauren E.¹,Bradbury Allison M.¹³,Gentry Atoska S.¹,Taylor Amanda R.⁴,Wooldridge Anne A.⁴,Wilhite Dewey R.³,Winter Randolph L.⁴,Whitlock Brian K.⁵,Johnson Jacob A.⁴,Holland Merilee⁴,Salibi Nouha⁶,Beyers Ronald J.⁷,Sartin James L.³,Denney Thomas S.⁷,Cox Nancy R.¹⁸,Sena-Esteves Miguel²,Martin Douglas R.¹³

Affiliation:

1. Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama

2. Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts

3. Department of Anatomy, Physiology, and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama

4. Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, Auburn, Alabama

5. Department of Large Animal Clinical Sciences, College of Veterinary Medicine, University of Tennessee, Knoxville, Tennessee

6. MR R&D Siemens Healthcare, Malvern, Pennsylvania.

7. Department of Electrical and Computer Engineering, Auburn University, Auburn, Alabama

8. Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, Alabama

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2017.163

Reference46 articles.

1. Prevalence of Lysosomal Storage Disorders

2. Natural History of Infantile GM2 Gangliosidosis

3. The Natural History of Juvenile or Subacute GM2 Gangliosidosis: 21 New Cases and Literature Review of 134 Previously Reported

4. Dramatically Different Phenotypes in Mouse Models of Human Tay-Sachs and Sandhoff Diseases

5. GM 2 Ganglioside Lysosomal Storage Disease in Cats with α-Hexosaminidase Deficiency

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