AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease-Reference-Cited by-同舟云学术

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

Published:2015-11-11 Issue:313 Volume:7 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Katz Martin L.¹,Tecedor Luis²,Chen Yonghong²,Williamson Baye G.³,Lysenko Elena²,Wininger Fred A.³,Young Whitney M.¹,Johnson Gayle C.⁴,Whiting Rebecca E. H.³,Coates Joan R.³,Davidson Beverly L.²⁵

Affiliation:

1. Department of Ophthalmology, University of Missouri, Columbia, MO 65212, USA.

2. The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

3. Department of Veterinary Medicine and Surgery, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, USA.

4. Department of Veterinary Pathobiology, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, USA.

5. Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

Abstract

AAV-mediated gene transfer to ependymal cells in a dog model of Batten disease provides sustained enzyme replacement and delays disease onset.

Funder

NIH

Roy J. Carver Trust

Batten Disease Support and Research Association

Children's Hospital of Philadelphia Research Institute

Jasper Against Batten Fund at Partnership for Cures

Blake's Purpose Foundation

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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2. Association of Mutations in a Lysosomal Protein with Classical Late-Infantile Neuronal Ceroid Lipofuscinosis

3. Biochemical Characterization of a Lysosomal Protease Deficient in Classical Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) and Development of an Enzyme-Based Assay for Diagnosis and Exclusion of LINCL in HUman Specimens and Animal Models

4. Gene therapy for lysosomal storage diseases

5. Distribution of extracellular tracers in perivascular spaces of the rat brain

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