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Titolo:
Gene therapy restores vision in a canine model of childhood blindness
Autore:
Acland, GM; Aguirre, GD; Ray, J; Zhang, Q; Aleman, TS; Cideciyan, AV; Pearce-Kelling, SE; Anand, V; Zeng, Y; Maguire, AM; Jacobson, SG; Hauswirth, WW; Bennett, J;
Indirizzi:
Univ Penn, Scheie Eye Inst, FM Kirby Ctr, Philadelphia, PA 19104 USA Univ Penn Philadelphia PA USA 19104 Kirby Ctr, Philadelphia, PA 19104 USA Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn Philadelphia PA USA 19104 phthalmol, Philadelphia, PA 19104 USA Univ Florida, Dept Ophthalmol, Gainesville, FL USA Univ Florida Gainesville FL USA da, Dept Ophthalmol, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida Gainesville FL USA ll Gene Therapy Ctr, Gainesville, FL USA
Titolo Testata:
NATURE GENETICS
fascicolo: 1, volume: 28, anno: 2001,
pagine: 92 - 95
SICI:
1061-4036(200105)28:1<92:GTRVIA>2.0.ZU;2-8
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT ADENOASSOCIATED VIRUS; VISUAL PIGMENT; RPE65; EXPRESSION; MUTATIONS; DYSTROPHY; RETINA;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
20
Recensione:
Indirizzi per estratti:
Indirizzo: Bennett, J Univ Penn, Scheie Eye Inst, FM Kirby Ctr, Philadelphia, PA 19104 USA Univ Penn Philadelphia PA USA 19104 Philadelphia, PA 19104 USA
Citazione:
G.M. Acland et al., "Gene therapy restores vision in a canine model of childhood blindness", NAT GENET, 28(1), 2001, pp. 92-95

Abstract

The relationship between the neurosensory photoreceptors and the adjacent retinal pigment epithelium (RPE) controls not only normal retinal function,but also the pathogenesis of hereditary retinal degenerations. The molecular bases for both primary photoreceptor(1) and RPE diseases(2-4) that causeblindness have been identified. Gene therapy has been used successfully toslow degeneration in rodent models of primary photoreceptor diseases(5,6),but efficacy of gene therapy directed at photoreceptors and RPE in a large-animal model of human disease has not been reported. Here we study one of the most clinically severe retinal degenerations, Leber congenital amaurosis (LCA). LCA causes near total blindness in infancy and can result from mutations in RPE65 (LCA. type II; MIM 180069 and 204100). A naturally occurring animal model, the RPE65(-/-) dog, suffers from early and severe visual impairment similar to that seen in human LCA. We used a recombinant adeno-associated virus (AAV) carrying wild-type RPE65 (AAV-RPE65) to test the efficacy of gene therapy in this model. Our results indicate that visual functionwas restored in this large animal model of childhood blindness.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 21/01/21 alle ore 02:52:45