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Progranulin Associates with Hexosaminidase A and Ameliorates GM2 Ganglioside Accumulation and Lysosomal Storage in Tay-Sachs Disease

Tay-Sachs disease (TSD) is a lethal lysosomal storage disease (LSD) caused by mutations in the HexA gene, which can lead to deficiency of β-hexosaminidase A (HexA) activity and consequent accumulation of its substrate, GM2 ganglioside. Recent reports that progranulin (PGRN) functions as a chaperone...

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Detalhes bibliográficos
Publicado no:J Mol Med (Berl)
Main Authors: Chen, Yuehong, Jian, Jinlong, Hettinghouse, Aubryanna, Zhao, Xueheng, Setchell, Kenneth D. R., Sun, Ying, Liu, Chuan-ju
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6240367/
https://ncbi.nlm.nih.gov/pubmed/30341570
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00109-018-1703-0
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