Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase.

To explore the possibility of using gene transfer to provide iduronate-2-sulfatase (IDS; EC 3.1.6.13) enzyme activity for treatment of Hunter syndrome, an amphotropic retroviral vector, L2SN, containing the human IDS coding sequence was constructed and studied for gene expression in vitro. Lymphobla...

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Bibliographic Details
Main Authors: Braun, S E, Aronovich, E L, Anderson, R A, Crotty, P L, McIvor, R S, Whitley, C B
Format: Artigo
Language:Inglês
Published: 1993
Subjects:
Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC48078/
https://ncbi.nlm.nih.gov/pubmed/8265633
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