Gene therapy for retinal disease.
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ABSTRACT:
SUBMITTER: McClements ME
PROVIDER: S-EPMC3831157 | biostudies-literature | 2013 Apr
REPOSITORIES: biostudies-literature
Publications
Gene therapy for retinal disease.
McClements Michelle E ME MacLaren Robert E RE
Translational research : the journal of laboratory and clinical medicine 20130108 4
Gene therapy strategies for the treatment of inherited retinal diseases have made major advances in recent years. This review focuses on adeno-associated viral (AAV) vector approaches to treat retinal degeneration and, thus, prevent or delay the onset of blindness. Data from human clinical trials of gene therapy for retinal disease show encouraging signs of safety and efficacy from AAV vectors. Recent progress in enhancing cell-specific targeting and transduction efficiency of the various retina ...[more]
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