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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
9
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pubmed:dateCreated |
2005-8-26
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pubmed:abstractText |
Retinitis pigmentosa GTPase regulator (RPGR) is a photoreceptor protein anchored in the connecting cilia by an RPGR-interacting protein (RPGRIP). Loss of RPGRIP causes Leber congenital amaurosis (LCA), a severe form of photoreceptor degeneration. The current study was an investigation of whether somatic gene replacement could rescue degenerating photoreceptors in a murine model of LCA due to a defect in RPGRIP.
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pubmed:grant |
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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 |
Sep
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pubmed:issn |
0146-0404
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pubmed:author |
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pubmed:issnType |
Print
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pubmed:volume |
46
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
3039-45
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pubmed:dateRevised |
2008-11-21
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pubmed:meshHeading |
pubmed-meshheading:16123399-Animals,
pubmed-meshheading:16123399-Blindness,
pubmed-meshheading:16123399-Dependovirus,
pubmed-meshheading:16123399-Disease Models, Animal,
pubmed-meshheading:16123399-Electroretinography,
pubmed-meshheading:16123399-Eye Proteins,
pubmed-meshheading:16123399-Fluorescent Antibody Technique, Indirect,
pubmed-meshheading:16123399-Gene Expression Regulation,
pubmed-meshheading:16123399-Gene Therapy,
pubmed-meshheading:16123399-Genetic Vectors,
pubmed-meshheading:16123399-Green Fluorescent Proteins,
pubmed-meshheading:16123399-Mice,
pubmed-meshheading:16123399-Mice, Knockout,
pubmed-meshheading:16123399-Photoreceptor Cells, Vertebrate,
pubmed-meshheading:16123399-Promoter Regions, Genetic,
pubmed-meshheading:16123399-Proteins,
pubmed-meshheading:16123399-Retinal Degeneration,
pubmed-meshheading:16123399-Rod Opsins,
pubmed-meshheading:16123399-Transfection
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pubmed:year |
2005
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pubmed:articleTitle |
Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.
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pubmed:affiliation |
Berman-Gund Laboratory for the Study of Retinal Degenerations, Harvard Medical School, Massachusetts Eye and Ear Infirmary, 243 Charles Street, Boston, MA 02114, 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,
Research Support, N.I.H., Extramural
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