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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...

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Detalles Bibliográficos
Autores principales:	Choi, Jin-Ok, Lee, Mi Hee, Park, Hae-Young, Jung, Sung-Chul
Formato:	Texto
Lenguaje:	English
Publicado:	BioMed Central 2010
Materias:	Research
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

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