Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow

The GM2 gangliosidoses are fatal lysosomal storage diseases principally affecting the brain. Absence of β-hexosaminidase A and B activities in the Sandhoff mouse causes neurological dysfunction and recapitulates the acute Tay–Sachs (TSD) and Sandhoff diseases (SD) in infants. Intracranial coinjectio...

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Main Authors: Cachón-González, M Begoña, Wang, Susan Z, McNair, Rosamund, Bradley, Josephine, Lunn, David, Ziegler, Robin, Cheng, Seng H, Cox, Timothy M
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2012
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3412498/
https://ncbi.nlm.nih.gov/pubmed/22453766
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.44
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