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Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer
BACKGROUND: Enzyme replacement therapy (ERT) with α-galactosidase A (α-Gal A) is currently the most effective therapeutic strategy for patients with Fabry disease, a lysosomal storage disease. However, ERT has limitations of a short half-life, requirement for frequent administration, and limited eff...
| Autores principales: | , , , |
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| Formato: | Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2010
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2861641/ https://www.ncbi.nlm.nih.gov/pubmed/20398385 http://dx.doi.org/10.1186/1423-0127-17-26 |