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Titolo:
Epitope-tagged recombinant AAV vectors for expressing neurturin and its receptor in retinal cells
Autore:
Jomary, C; Grist, J; Milbrandt, J; Neal, M; Jones, S;
Indirizzi:
Univ London Kings Coll, Div Pharmacol & Therapeut, Retinitis Pigmentosa Res Unit,St Thomas Hosp, Guys Kings & St Thomas Sch Biomed Sci,Rayne Inst, London SE1 7EH, England Univ London Kings Coll London England SE1 7EH t, London SE1 7EH, England Washington Univ, Sch Med, Dept Internal Med, St Louis, MO 63110 USA Washington Univ St Louis MO USA 63110 nternal Med, St Louis, MO 63110 USA Washington Univ, Sch Med, Dept Pathol, Div Lab Med, St Louis, MO 63110 USAWashington Univ St Louis MO USA 63110 Div Lab Med, St Louis, MO 63110 USA
Titolo Testata:
MOLECULAR VISION
fascicolo: 6, volume: 7, anno: 2001,
pagine: 36 - 41
SICI:
1090-0535(20010219)7:6<36:ERAVFE>2.0.ZU;2-H
Fonte:
ISI
Lingua:
ENG
Soggetto:
MEDIATED GENE-TRANSFER; PHOTORECEPTOR DEGENERATION; NEUROTROPHIC FACTOR; TRANSGENIC MICE; ROD PHOTORECEPTORS; REPORTER GENE; MOUSE RETINA; RD MOUSE; IN-VITRO; RHODOPSIN;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
29
Recensione:
Indirizzi per estratti:
Indirizzo: Jomary, C Univ London Kings Coll, Div Pharmacol & Therapeut, Retinitis Pigmentosa Res Unit,St Thomas Hosp, Guys Kings & St Thomas Sch Biomed Sci,Rayne Inst, London SE1 7EH, England Univ London Kings Coll London England SE17EH SE1 7EH, England
Citazione:
C. Jomary et al., "Epitope-tagged recombinant AAV vectors for expressing neurturin and its receptor in retinal cells", MOL VIS, 7(6), 2001, pp. 36-41

Abstract

Purpose: Neurturin (NTN) is a potent neuronal survival factor in the central and peripheral nervous systems. We previously described altered expression of mRNAs for NTN and one of its receptor components, GFR alpha -2 in degenerative retinas of rd/rd mice. Towards assessing the potential for transfer of these genes to counteract retinal degeneration, we examined recombinant adeno-associated virus (rAAV) constructs for expression of NTN and GFR alpha -2 transgenes in retinal cells in vitro and for the effect of transgene expression on retinal function following intraocular delivery in rd/rd mice. Methods: The rAAV constructs incorporated epitope tags to facilitate discrimination between transgenic and endogenous expression. Expression of murine NTN was driven by a CMV promoter and a partial murine opsin promoter was used to drive expression of human GFR alpha -2. rAAV preparations were usedto infect mouse retinal cell cultures and for intraocular injection of predegenerative rd/rd mice. Endogenous and transgene expression was analyzed by immunofluorescence. Photoreceptor function in treated mice was assessed by electroretinography. Results: Both vectors delivered and expressed their transgenes in vitro and in vivo. Differential targeting was achieved in vivo through the use of alternative promoters. Under the conditions examined, no functional rescue of rd photoreceptors was observed. Conclusions: Therapeutic treatment of the rd model of retinal degenerationdoes not appear to be effected by simple modulation of the expression of NTN or GFR alpha -2, and may therefore depend on additional synergistic factors. Our AAV constructs will facilitate the development of combinatorial approaches to the treatment of central and peripheral neurodegenerations.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 22/01/20 alle ore 06:50:19