Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy

Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy

GM1 gangliosidosis is a fatal lysosomal disorder, for which there is no effective treatment. Adeno-associated virus (AAV) gene therapy in GM1 cats has resulted in a greater than 6-fold increase in lifespan, with many cats remaining alive at >5.7 years of age, with minimal clinical signs. Glycolip...

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Bibliographic Details
Main Authors: Heather L. Gray-Edwards, Xuntian Jiang, Ashley N. Randle, Amanda R. Taylor, Taylor L. Voss, Aime K. Johnson, Victoria J. McCurdy, Miguel Sena-Esteves, Daniel S. Ory, Douglas R. Martin
Format: Article
Language:English
Published: Elsevier 2017-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
adeno-associated virus
AAV
biomarkers
feline
gene therapy
GM1 gangliosidosis
lipidomics
neurologic disease
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300888

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http://www.sciencedirect.com/science/article/pii/S2329050117300888

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