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Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

Investigative ophthalmology & visual science - 1 Apr 2003

Narfström Kristina, Katz Martin L, Bragadottir Ragnheidur, Seeliger Mathias, Boulanger Ana, Redmond T Michael, Caro Lynette, Lai Chooi-May, Rakoczy P Elizabeth

Abstract excerpt

PURPOSE: To assess the efficacy of AAV-mediated gene therapy to restore vision in a large number of RPE65(-/-) dogs and to determine whether systemic and local side effects are caused by the treatment. METHODS: Normal RPE65 dog cDNA was subcloned into an rAAV vector under control of a cytomegalovirus promoter, and an AAV.GFP control vector was also produced with the titers 2 x 10(12) particles/mL and 2 x 10(10)...

Topics

Animals
Dark Adaptation
Dependovirus
Dog Diseases
Dogs
Electroretinography
Eye Proteins
Female
Gene Transfer Techniques
Genetic Therapy
Genetic Vectors

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Green Fluorescent Proteins

Indicators and Reagents

Lipid Metabolism

Luminescent Proteins

Male

Mutation

Night Blindness

Source

Identifiers

PMID: 12657607
DOI: 10.1167/iovs.02-0595

Genes

RPE65
RPE
ERG

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.PMID 12657607Read ↗

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