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Article

Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer.

Proceedings of the National Academy of Sciences of the United States of America - 15 Aug 1995

Li T, Davidson B L

Abstract excerpt

We have studied the use of adenovirus-mediated gene transfer to reverse the pathologic changes of lysosomal storage disease caused by beta-glucuronidase deficiency in the eyes of mice with mucopolysaccharidosis VII. A recombinant adenovirus carrying the human beta-glucuronidase cDNA coding region under the control of a non-tissue-specific promoter was injected intravitreally or subretinally into the eyes of mice...

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Identifiers

PMID: 7644479
DOI: 10.1073/pnas.92.17.7700
PMCID: PMC41213

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RPE

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