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

doi:10.1073/pnas.92.17.7700

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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...

Topics

Adenoviridae
Animals
DNA, Complementary
Gene Expression
Gene Transfer Techniques
Genetic Therapy
Glucuronidase
Humans
In Situ Hybridization
Mice
Mice, Inbred BALB C

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Mice, Inbred C57BL

Mice, Mutant Strains

Mucopolysaccharidosis VII

Phenotype

Pigment Epithelium of Eye

Reference Values

beta-Galactosidase

Source

Open in PubMed Open full text

Identifiers

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

Genes

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 29 Aug 2026. Prepared record publication-7644479-2026-08-29.1. Manifest publication-graph-cohort-2026-08-29.1.

Source receipt: PubMed via Helena Literature Corpus, retrieved 29 Aug 2026.

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