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Titolo:
Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa
Autore:
Sanftner, LHM; Abel, H; Hauswirth, WW; Flannery, JG;
Indirizzi:
Univ Calif Berkeley, Dept Vis Sci, Berkeley, CA 94720 USA Univ Calif Berkeley Berkeley CA USA 94720 Vis Sci, Berkeley, CA 94720 USA Univ Calif Berkeley, Neurosci Grp, Berkeley, CA 94720 USA Univ Calif Berkeley Berkeley CA USA 94720 sci Grp, Berkeley, CA 94720 USA Univ Florida, Dept Mol Genet, Gainesville, FL 32610 USA Univ Florida Gainesville FL USA 32610 ol Genet, Gainesville, FL 32610 USA Univ Florida, Dept Microbiol & Ophthalmol, Gainesville, FL 32610 USA Univ Florida Gainesville FL USA 32610 hthalmol, Gainesville, FL 32610 USA Univ Florida, Powel Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida Gainesville FL USA 32610 rapy Ctr, Gainesville, FL 32610 USA
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 6, volume: 4, anno: 2001,
pagine: 622 - 629
SICI:
1525-0016(200112)4:6<622:GCLDNF>2.0.ZU;2-Z
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT ADENOASSOCIATED VIRUS; FIBROBLAST GROWTH-FACTOR; RETINAL DEGENERATION; GENE-THERAPY; DOPAMINERGIC-NEURONS; TYROSINE KINASE; LIGHT DAMAGE; RD MICE; IN-VIVO; GDNF;
Keywords:
rAAV vector; gene therapy; retinal degeneration; GDNF;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
39
Recensione:
Indirizzi per estratti:
Indirizzo: Flannery, JG Univ Calif Berkeley, Dept Vis Sci, Berkeley, CA 94720 USA Univ Calif Berkeley Berkeley CA USA 94720 eley, CA 94720 USA
Citazione:
L.H.M. Sanftner et al., "Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa", MOL THER, 4(6), 2001, pp. 622-629

Abstract

We designed experiments to evaluate the therapeutic potential of glial cell line derived neurotrophic factor (GDNF) to rescue photoreceptors from genetically determined cell death. Gene transfer of the neurotrophic factor tothe retina was achieved via a recombinant adeno-associated virus (rAAV) vector containing the chicken beta -actin promoter/immediate early cytomegalovirus enhancer (CBA) driving the human GDNF gene. We delivered AAV-CBA-GDNFto the retinas of an animal model of retinitis pigmentosa, the TgN S334ter-4 rhodopsin line of transgenic rats. Immunohistochemical studies localizedAAV-CBA-GDNF-derived recombinant protein to cell bodies, inner segments, and outer segments of photoreceptor cells as well as to retinal pigment epithelial cells. We assessed the effect of viral delivery by morphometric and electroretinographic analysis. These experiments showed that GDNF vector treatment leads to increased rod photoreceptor survival as indicated by morphometric analysis of outer nuclear layer thickness. AAV-CBA-GDNF-treated retinas also demonstrated functional improvement by the substantially increased amplitude of electroretinograms. AAV-CBA-GDNF delivery had a significant rescue effect on photoreceptor degeneration in this animal model.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 20/01/20 alle ore 04:48:13