Sustained Normalization of Neurological Disease after Intracranial Gene Therapy in a Feline Model-Reference-Cited by-同舟云学术

Sustained Normalization of Neurological Disease after Intracranial Gene Therapy in a Feline Model

Published:2014-04-09 Issue:231 Volume:6 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

McCurdy Victoria J.¹²,Johnson Aime K.³,Gray-Edwards Heather L.¹,Randle Ashley N.¹,Brunson Brandon L.²,Morrison Nancy E.¹,Salibi Nouha⁴⁵,Johnson Jacob A.³,Hwang Misako¹,Beyers Ronald J.⁵,Leroy Stanley G.⁶,Maitland Stacy⁶,Denney Thomas S.⁵⁷,Cox Nancy R.¹⁸,Baker Henry J.¹⁸,Sena-Esteves Miguel⁶,Martin Douglas R.¹²

Affiliation:

1. Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA.

2. Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA.

3. Department of Clinical Sciences, Auburn College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA.

4. MR R&D, Siemens Healthcare, Malvern, PA 19355, USA.

5. Auburn University MRI Research Center, Auburn University, Auburn, AL 36849, USA.

6. Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA.

7. Department of Electrical and Computer Engineering, Auburn University, Auburn, AL 36849, USA.

8. Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, USA.

Abstract

In a feline model of lysosomal storage disease, intracranial gene therapy achieved therapeutic efficacy in the CNS and increased long-term survival.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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