7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy

7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy

GM1 gangliosidosis (GM1) is a fatal neurodegenerative lysosomal storage disease that occurs most commonly in young children, with no effective treatment available. Long-term follow-up of GM1 cats treated by bilateral thalamic and deep cerebellar nuclei (DCN) injection of adeno-associated virus (AAV)...

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Bibliographic Details
Main Authors: Heather L. Gray-Edwards, Anne S. Maguire, Nouha Salibi, Lauren E. Ellis, Taylor L. Voss, Elise B. Diffie, Jey Koehler, Ashley N. Randle, Amanda R. Taylor, Brandon L. Brunson, Thomas S. Denney, Ronald J. Beyers, Atoska S. Gentry, Amanda L. Gross, Ana R. Batista, Miguel Sena-Esteves, Douglas R. Martin
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gangliosidosis
GM1
lysosomal storage disease
adeno-associated virus
AAV
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050119301536

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http://www.sciencedirect.com/science/article/pii/S2329050119301536

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