Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan

GM1 gangliosidosis (GM1) is an autosomal recessive lysosomal storage disease where GLB1 gene mutations result in a reduction or absence of lysosomal acid β-galactosidase (βgal) activity. βgal deficiency leads to accumulation of GM1-ganglioside in the central nervous system (CNS). GM1 is characterize...

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Publicado en:Hum Mol Genet
Main Authors: Weismann, Cara M., Ferreira, Jennifer, Keeler, Allison M., Su, Qin, Qui, Linghua, Shaffer, Scott A., Xu, Zuoshang, Gao, Guangping, Sena-Esteves, Miguel
Formato: Artigo
Idioma:Inglês
Publicado: Oxford University Press 2015
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Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4492398/
https://ncbi.nlm.nih.gov/pubmed/25964428
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv168
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