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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Bibliografiska uppgifter
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: 1990
Ämnen:
Research Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC53796/
https://ncbi.nlm.nih.gov/pubmed/2158095
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