AAV-Mediated Gene Delivery in a Feline Model of Sandhoff Disease Corrects Lysosomal Storage in the Central Nervous System

AAV-Mediated Gene Delivery in a Feline Model of Sandhoff Disease Corrects Lysosomal Storage in the Central Nervous System

Sandhoff disease (SD) is an autosomal recessive neurodegenerative disease caused by a mutation in the gene for the β-subunit of β-N-acetylhexosaminidase (Hex), resulting in the inability to catabolize ganglioside GM2 within the lysosomes. SD presents with an accumulation of GM2 and its asialo deriva...

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Bibliographic Details
Main Authors: Hannah E. Rockwell, Victoria J. McCurdy, Samuel C. Eaton, Diane U. Wilson, Aime K. Johnson, Ashley N. Randle, Allison M. Bradbury, Heather L. Gray-Edwards, Henry J. Baker, Judith A. Hudson, Nancy R. Cox, Miguel Sena-Esteves, Thomas N. Seyfried, Douglas R. Martin
Format: Article
Language:English
Published: SAGE Publishing 2015-04-01
Series:ASN Neuro
Online Access:https://doi.org/10.1177/1759091415569908

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https://doi.org/10.1177/1759091415569908

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