Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease

The GM2 gangliosidoses, Tay-Sachs disease (TSD) and Sandhoff disease (SD), are progressive neurodegenerative disorders that are caused by a mutation in the enzyme β-N-acetylhexosaminidase (Hex). Due to the recent emergence of novel experimental treatments, biomarker development has become particular...

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Detalhes bibliográficos
Publicado no:Exp Neurol
Main Authors: Bradbury, Allison M, Gray-Edwards, Heather L, Shirley, Jamie L, McCurdy, Victoria J, Colaco, Alexandria N, Randle, Ashley N, Christopherson, Pete W, Bird, Allison C, Johnson, Aime K, Wilson, Diane U, Hudson, Judith A, De Pompa, Nicholas L, Sorjonen, Donald C, Brunson, Brandon L, Jeyakumar, Mylvaganam, Platt, Frances M, Baker, Henry J, Cox, Nancy R, Sena-Esteves, Miguel, Martin, Douglas R
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4262540/
https://ncbi.nlm.nih.gov/pubmed/25284324
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.expneurol.2014.09.020
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