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PredicateObject
rdf:type
lifeskim:mentions
pubmed:dateCreated
2010-3-18
pubmed:abstractText
Recombinant adeno-associated viral (rAAV) vectors have recently been widely used for the delivery of therapeutic transgenes in preclinical and clinical studies for inherited retinal degenerative diseases. Interchanging capsid genes between different AAV serotypes has enabled selective delivery of transgene into specific cell type(s) of the retina. The RP10 form of autosomal dominant retinitis pigmentosa (adRP) is caused by missense mutations within the gene encoding inosine 5'-monophosphate dehydrogenase type 1. Here, we report that the use of rAAV2/5 vectors expressing shRNA targeting mutant IMPDH1 prevents photoreceptor degeneration, and preserves synaptic connectivity in a mouse model of RP10.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:issn
0065-2598
pubmed:author
pubmed:issnType
Print
pubmed:volume
664
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
559-65
pubmed:meshHeading
pubmed:year
2010
pubmed:articleTitle
Protection of photoreceptors in a mouse model of RP10.
pubmed:affiliation
Department of Genetics, Trinity College Dublin, Dublin 2, Ireland. lawrenct@tcd.ie
pubmed:publicationType
Journal Article, Research Support, Non-U.S. Gov't