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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Detalhes bibliográficos
Publicado no:ASN Neuro
Main Authors: Rockwell, Hannah E., McCurdy, Victoria J., Eaton, Samuel C., Wilson, Diane U., Johnson, Aime K., Randle, Ashley N., Bradbury, Allison M., Gray-Edwards, Heather L., Baker, Henry J., Hudson, Judith A., Cox, Nancy R., Sena-Esteves, Miguel, Seyfried, Thomas N., Martin, Douglas R.
Formato: Artigo
Idioma:Inglês
Publicado em: SAGE Publications 2015
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4720176/
https://ncbi.nlm.nih.gov/pubmed/25873306
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1759091415569908
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