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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
13
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pubmed:dateCreated |
1997-7-21
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pubmed:abstractText |
We describe a general approach for achieving efficient and cell type-specific expression of exogenous genes in photoreceptor cells of the mammalian retina. Recombinant adeno-associated virus (rAAV) vectors were used to transfer the bacterial lacZ gene or a synthetic green fluorescent protein gene (gfp) to mouse or rat retinas after injection into the subretinal space. Using a proximal murine rod opsin promoter (+86 to -385) to drive expression, reporter gene product was found exclusively in photoreceptors, not in any other retinal cell type or in the adjacent retinal pigment epithelium. GFP-expressing photoreceptors typically encompassed 10-20% of the total retinal area after a single 2-microl injection. Photoreceptors were transduced with nearly 100% efficiency in the region directly surrounding the injection site. We estimate approximately 2.5 million photoreceptors were transduced as a result of the single subretinal inoculation. This level of gene transfer and expression suggests the feasibility of genetic therapy for retinal disease. The gfp-containing rAAV stock was substantially free of both adenovirus and wild-type AAV, as judged by plaque assay and infectious center assay, respectively. Thus, highly purified, helper virus-free rAAV vectors can achieve high-frequency tissue-specific transduction of terminally differentiated, postmitotic photoreceptor cells.
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pubmed:grant |
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pubmed:commentsCorrections |
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-1316261,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-1350091,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-1358680,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-1385966,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-1825171,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-1827795,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-1899580,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-2168521,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-2324310,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-2835501,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-2914751,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-3102226,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-6256308,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-7479749,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-7504271,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-7584067,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-7733180,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-7777575,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-7812140,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-7842013,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8090744,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8106474,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8163342,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8163343,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8523565,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8550318,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8627709,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8627803,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8627804,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8640555,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8676491,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8692941,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8733124,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8892935,
http://linkedlifedata.com/resource/pubmed/commentcorrection/9192666-8943064
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
IM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Jun
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pubmed:issn |
0027-8424
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pubmed:author |
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pubmed:issnType |
Print
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pubmed:day |
24
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pubmed:volume |
94
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
6916-21
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pubmed:dateRevised |
2009-11-18
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pubmed:meshHeading |
pubmed-meshheading:9192666-Animals,
pubmed-meshheading:9192666-DNA, Recombinant,
pubmed-meshheading:9192666-Dependovirus,
pubmed-meshheading:9192666-Gene Expression,
pubmed-meshheading:9192666-Gene Transfer Techniques,
pubmed-meshheading:9192666-Genetic Vectors,
pubmed-meshheading:9192666-Lac Operon,
pubmed-meshheading:9192666-Mice,
pubmed-meshheading:9192666-Mice, Inbred C57BL,
pubmed-meshheading:9192666-Photoreceptor Cells,
pubmed-meshheading:9192666-Rats,
pubmed-meshheading:9192666-Rats, Sprague-Dawley,
pubmed-meshheading:9192666-Retina
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pubmed:year |
1997
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pubmed:articleTitle |
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus.
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pubmed:affiliation |
School of Optometry and Neuroscience Group, University of California, Berkeley, CA 94720, USA.
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pubmed:publicationType |
Journal Article,
Research Support, U.S. Gov't, P.H.S.,
Research Support, Non-U.S. Gov't
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