Á lódáil...
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...
Na minha lista:
| Foilsithe in: | Mol Ther |
|---|---|
| Main Authors: | , , , , , , , , , , , |
| Formáid: | Artigo |
| Teanga: | Inglês |
| Foilsithe: |
Nature Publishing Group
2015
|
| Ábhair: | |
| Rochtain Ar Líne: | 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 |
| Clibeanna: |
Cuir Clib Leis
Gan Chlibeanna, Bí ar an gcéad duine leis an taifead seo a chlibeáil!
|