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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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Udgivet i: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
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 1990
Fag:
Research Article
Online adgang: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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