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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...
Tallennettuna:
| Julkaisussa: | Mol Ther |
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| Päätekijät: | , , , , , , , , , , , |
| Aineistotyyppi: | Artigo |
| Kieli: | Inglês |
| Julkaistu: |
Nature Publishing Group
2015
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| Aiheet: | |
| Linkit: | 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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