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Predicate | Object |
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rdf:type | |
lifeskim:mentions | |
pubmed:issue |
7
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pubmed:dateCreated |
1997-9-19
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pubmed:abstractText |
Knowledge of the mutations leading to inherited retinal degenerations provides a foundation for the development of somatic gene therapy in which potentially corrective genes are transferred to the target photoreceptor cells. Towards this end, we have evaluated the efficacy of a recombinant adeno-associated virus (AAV) vector to deliver and express the correct form of the cGMP phosphodiesterase-beta (PDE-beta) gene in the retinas of rd mice, which suffer rapid retinal degeneration due to recessive mutation in the endogenous gene. A truncated murine opsin promoter was used to drive expression of the PDE-beta cDNA. Following intraocular injection of AAV. PDE-beta, increased retinal expression of immunoreactive PDE protein was observed, including within photoreceptor cell bodies. Compared with age-matched controls, treated eyes showed increased numbers of photoreceptors and a two-fold increase in sensitivity to light as measured by in vitro electroretinography. These findings provide evidence that rescue of functional photoreceptor neurons can be achieved by somatic gene therapy.
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pubmed:language |
eng
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pubmed:journal | |
pubmed:citationSubset |
IM
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pubmed:status |
MEDLINE
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pubmed:month |
Jul
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pubmed:issn |
0969-7128
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pubmed:author | |
pubmed:issnType |
Print
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pubmed:volume |
4
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
683-90
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pubmed:dateRevised |
2008-11-21
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pubmed:meshHeading |
pubmed-meshheading:9282169-Animals,
pubmed-meshheading:9282169-Dependovirus,
pubmed-meshheading:9282169-Disease Models, Animal,
pubmed-meshheading:9282169-Electroretinography,
pubmed-meshheading:9282169-Gene Transfer Techniques,
pubmed-meshheading:9282169-Genetic Vectors,
pubmed-meshheading:9282169-Mice,
pubmed-meshheading:9282169-Mice, Inbred C57BL,
pubmed-meshheading:9282169-Mice, Mutant Strains,
pubmed-meshheading:9282169-Photoreceptor Cells,
pubmed-meshheading:9282169-Retinal Degeneration
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pubmed:year |
1997
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pubmed:articleTitle |
Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration.
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pubmed:affiliation |
British Retinitis Pigmentosa Society Laboratory, Department of Pharmacology, UMDS, Rayne Institute, St Thomas' Hospital, London, UK.
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pubmed:publicationType |
Journal Article,
Research Support, Non-U.S. Gov't
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