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Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis
Adeno-associated virus (AAV) mediated gene replacement for lysosomal disorders have been spurred by the ability of some serotypes to efficiently transduce neurons in the brain and by the ability of lysosomal enzymes to cross-correct among cells. Here, we explored enzyme replacement therapy in a knoc...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
2011
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3235265/ https://www.ncbi.nlm.nih.gov/pubmed/21900963 http://dx.doi.org/10.1038/gt.2011.118 |