Restoration of normal lysosomal function in mucopolysaccharidosis type VII cells by retroviral vector-mediated gene transfer.

Retroviral vectors were constructed containing a rat beta-glucuronidase cDNA driven by heterologous promoters. Vector-mediated gene transfer into human and canine beta-glucuronidase-deficient mucopolysaccharidosis type VII fibroblasts completely corrected the deficiency in beta-glucuronidase enzymat...

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Detalles Bibliográficos

Publicado en:	Proc Natl Acad Sci U S A
Principais autores:	Wolfe, J H, Schuchman, E H, Stramm, L E, Concaugh, E A, Haskins, M E, Aguirre, G D, Patterson, D F, Desnick, R J, Gilboa, E
Formato:	Artigo
Idioma:	Inglês
Publicado:	National Academy of Sciences 1990
Assuntos:	Research Article
Acceso en liña:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC53796/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/2158095/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.87.8.2877
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