Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain-Reference-Cited by-同舟云学术

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

Published:2017-06 Issue:6 Volume:28 Page:510-522
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Golebiowski Diane¹²,van der Bom Imramsjah M.J.³⁴,Kwon Churl-Su⁵,Miller Andrew D.⁶,Petrosky Keiko⁶,Bradbury Allison M.⁷⁸,Maitland Stacy¹²,Kühn Anna Luisa³⁴,Bishop Nina⁹,Curran Elizabeth⁶,Silva Nilsa⁶,GuhaSarkar Dwijit¹²,Westmoreland Susan V.⁶,Martin Douglas R.⁷⁸,Gounis Matthew J.³⁴,Asaad Wael F.¹⁰¹¹¹²,Sena-Esteves Miguel¹²

Affiliation:

1. Department of Neurology, University of Massachusetts Medical School, Worcester, Massachusetts

2. Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts

3. Department of Radiology, University of Massachusetts Medical School, Worcester, Massachusetts

4. New England Center for Stroke Research, University of Massachusetts Medical School, Worcester, Massachusetts

5. Department of Neurosurgery, Massachusetts General Hospital, Boston, Massachusetts

6. New England Primate Research Center, Harvard Medical School, Southborough, Massachusetts

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

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

9. Department of Animal Medicine, University of Massachusetts Medical School, Worcester, Massachusetts

10. Department of Neurosurgery, Alpert Medical School, Brown University, Providence, Rhode Island

11. Brown Institute for Brain Science, Brown University, Providence, Rhode Island

12. Rhode Island Hospital, Providence, Rhode Island.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.109

Reference66 articles.

1. Natural History of Infantile GM2 Gangliosidosis

2. Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges

3. Central nervous system therapy for lysosomal storage disorders

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