Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates

G(M2) gangliosidoses are severe neurodegenerative disorders resulting from a deficiency in β-hexosaminidase A activity and lacking effective therapies. Using a Sandhoff disease (SD) mouse model (Hexb(−/−)) of the G(M2) gangliosidoses, we tested the potential of systemically delivered adeno-associate...

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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: Walia, Jagdeep S, Altaleb, Naderah, Bello, Alexander, Kruck, Christa, LaFave, Matthew C, Varshney, Gaurav K, Burgess, Shawn M, Chowdhury, Biswajit, Hurlbut, David, Hemming, Richard, Kobinger, Gary P, Triggs-Raine, Barbara
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2015
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4351464/
https://ncbi.nlm.nih.gov/pubmed/25515709
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.240
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