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Effective gene therapy in an authentic model of Tay-Sachs-related diseases

Tay-Sachs disease is a prototypic neurodegenerative disease. Lysosomal storage of GM2 ganglioside in Tay-Sachs and the related disorder, Sandhoff disease, is caused by deficiency of β-hexosaminidase A, a heterodimeric protein. Tay-Sachs-related diseases (GM2 gangliosidoses) are incurable, but gene t...

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Main Authors: Cachón-González, M. Begoña, Wang, Susan Z., Lynch, Andrew, Ziegler, Robin, Cheng, Seng H., Cox, Timothy M.
Formato: Artigo
Idioma:Inglês
Publicado: National Academy of Sciences 2006
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC1482797/
https://ncbi.nlm.nih.gov/pubmed/16801539
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0603765103
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