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Titolo:
An allele-specific hammerhead ribozyme gene therapy for a porcine model ofautosomal dominant retinitis pigmentosa
Autore:
Shaw, LC; Skold, A; Wong, F; Petters, R; Hauswirth, W; Lewin, AS;
Indirizzi:
Univ Florida, Coll Med, Dept Mol Genet & Microbiol, Gainesville, FL 32610 USA Univ Florida Gainesville FL USA 32610 icrobiol, Gainesville, FL 32610 USA Univ Florida, Coll Med, Dept Pharmacol & Therapeut, Gainesville, FL 32610 USA Univ Florida Gainesville FL USA 32610 herapeut, Gainesville, FL 32610 USA Univ Florida, Coll Med, Dept Ophthalmol, Gainesville, FL 32610 USA Univ Florida Gainesville FL USA 32610 hthalmol, Gainesville, FL 32610 USA Duke Univ, Sch Med, Dept Ophthalmol, Durham, NC USA Duke Univ Durham NC USA e Univ, Sch Med, Dept Ophthalmol, Durham, NC USA Duke Univ, Sch Med, Dept Pathol, Durham, NC USA Duke Univ Durham NC USADuke Univ, Sch Med, Dept Pathol, Durham, NC USA N Carolina State Univ, Dept Anim Sci, Raleigh, NC 27695 USA N Carolina State Univ Raleigh NC USA 27695 nim Sci, Raleigh, NC 27695 USA
Titolo Testata:
MOLECULAR VISION
fascicolo: 2, volume: 7, anno: 2001,
pagine: 6 - 13
SICI:
1090-0535(20010126)7:2<6:AAHRGT>2.0.ZU;2-O
Fonte:
ISI
Lingua:
ENG
Soggetto:
RETINAL DEGENERATION; RHODOPSIN MUTATION; RNA; VIRUS; CELLS;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
34
Recensione:
Indirizzi per estratti:
Indirizzo: Lewin, AS Univ Florida, Coll Med, Dept Mol Genet & Microbiol, Box 100266, Gainesville, FL 32610 USA Univ Florida Box 100266 Gainesville FL USA 32610 e, FL 32610 USA
Citazione:
L.C. Shaw et al., "An allele-specific hammerhead ribozyme gene therapy for a porcine model ofautosomal dominant retinitis pigmentosa", MOL VIS, 7(2), 2001, pp. 6-13

Abstract

PURPOSE: To develop a hammerhead ribozyme-based gene therapy for a porcinemodel of autosomal dominant retinitis pigmentosa (ADRP). METHODS: Hammerhead ribozymes were developed and assayed in vitro against RNA targets homologous to the opsin P347S mutants found in a transgenic porcine model and in humans. Both cloned and synthetic RNA oligonucleotide versions of ribozymes and targets were tested under multiple-turnover conditions using oligonucleotide RNA targets. Digestion of full-length P347S mRNA from porcine retina was performed. RESULTS: The porcine P347S hammerhead ribozyme was specific for the opsin P347S sequence. Multiple-turnover analysis yielded the following kinetic parameters: V-max=7.3 +/-0.5 nM/min, K-m=2.1 +/-0.6 mM, and k(cat)=1.5 +/-0.4min(-1). The human P347S hammerhead ribozyme was substantially less active(similar to 10,000 fold). CONCLUSIONS: We have developed a hammerhead ribozyme to use as a model forgene therapy of autosomal dominant retinitis pigmentosa in a transgenic porcine model. Based on kinetic characterization of this ribozyme compared toothers used for gene therapy, this should be an effective reagent RNA. Theallele specific ribozyme we tested for the human sequence, however, is notlikely to be useful for gene therapy indicating that an alternative approach is necessary.

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Documento generato il 23/01/20 alle ore 18:19:23