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Titolo:
Gene therapy and retinitis pigmentosa: advances and future challenges
Autore:
Dejneka, NS; Bennett, J;
Indirizzi:
Univ Penn, Scheie Eye Inst, Stellar Chance Labs 310, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Univ Penn Philadelphia PA USA 19104 phthalmol, Philadelphia, PA 19104 USA
Titolo Testata:
BIOESSAYS
fascicolo: 7, volume: 23, anno: 2001,
pagine: 662 - 668
SICI:
0265-9247(200107)23:7<662:GTARPA>2.0.ZU;2-6
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT ADENOASSOCIATED VIRUS; MEDIATED RETINAL TRANSDUCTION; CILIARY NEUROTROPHIC FACTOR; PHOTORECEPTOR CELL-DEATH; IN-VIVO; REPORTER GENE; TRANSGENE EXPRESSION; ROD PHOTORECEPTORS; LENTIVIRAL VECTOR; MOUSE RETINA;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
70
Recensione:
Indirizzi per estratti:
Indirizzo: Bennett, J Univ Penn, Scheie Eye Inst, Stellar Chance Labs 310, FM Kirby Ctr Mol Ophthalmol, 422 Curie Blvd, Philadelphia, PA 19104 USA Univ Penn 422Curie Blvd Philadelphia PA USA 19104 PA 19104 USA
Citazione:
N.S. Dejneka e J. Bennett, "Gene therapy and retinitis pigmentosa: advances and future challenges", BIOESSAYS, 23(7), 2001, pp. 662-668

Abstract

It may be possible, one day, to use gene therapy to treat diseases whose genetic defects have been discerned. Because many genes responsible for inherited eye disorders within the retina have been identified, diseases of theeye are prime candidates for this form of therapy. The eye also has the advantage of being highly accessible with altered immunological properties, important considerations for easy delivery of virus and avoidance of systemic immune responses. Currently, adenovirus, adeno-associated virus and lentivirus have been used to successfully transfer genetic material to retinal pigment epithelium and photoreceptor cells. By harnessing therapeutic genes to these viruses, researchers have been able to demonstrate rescue in rodent models of retinitis pigmentosa, providing evidence that this form of therapy can be effective in delaying photoreceptor cell death. Future challenges include confirming therapeutic effects in animal models with eyes more anatomically similar to those of humans and demonstrating long-term rescue with minimal toxicity. BioEssays 23:662-668, 2001. (C) 2001 John Wiley & Sons, Inc.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 23/01/21 alle ore 10:17:07