Protection of photoreceptors in a mouse model of RP10.
Adv Exp Med Biol
; 664: 559-65, 2010.
Article
em En
| MEDLINE
| ID: mdl-20238059
ABSTRACT
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.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Retinose Pigmentar
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Citoproteção
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Células Fotorreceptoras de Vertebrados
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Modelos Animais de Doenças
Limite:
Animals
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Humans
Idioma:
En
Ano de publicação:
2010
Tipo de documento:
Article