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Titolo:
Full functional rescue of a complete muscle (TA) in dystrophic hamsters byadeno-associated virus vector-directed gene therapy
Autore:
Xiao, X; Li, J; Tsao, YP; Dressman, D; Hoffman, EP; Watchko, JF;
Indirizzi:
Univ Pittsburgh, Dept Mol Genet & Biochem, Sch Med, Pittsburgh, PA 15261 USA Univ Pittsburgh Pittsburgh PA USA 15261 Sch Med, Pittsburgh, PA 15261 USA Univ Pittsburgh, Duchenne Muscular Dystrophy Res Ctr, Sch Med, Pittsburgh,PA 15261 USA Univ Pittsburgh Pittsburgh PA USA 15261 Sch Med, Pittsburgh,PA 15261 USA Univ Pittsburgh, Dept Pediat, Sch Med, Pittsburgh, PA 15261 USA Univ Pittsburgh Pittsburgh PA USA 15261 Sch Med, Pittsburgh, PA 15261 USA Univ Pittsburgh, Magee Womens Res Inst, Sch Med, Pittsburgh, PA 15261 USA Univ Pittsburgh Pittsburgh PA USA 15261 Sch Med, Pittsburgh, PA 15261 USA Natl Def Med Ctr, Taipei, Taiwan Natl Def Med Ctr Taipei TaiwanNatl Def Med Ctr, Taipei, Taiwan Childrens Natl Med Ctr, Res Ctr Genet Med, Washington, DC 20010 USA Childrens Natl Med Ctr Washington DC USA 20010 , Washington, DC 20010 USA
Titolo Testata:
JOURNAL OF VIROLOGY
fascicolo: 3, volume: 74, anno: 2000,
pagine: 1436 - 1442
SICI:
0022-538X(200002)74:3<1436:FFROAC>2.0.ZU;2-4
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT ADENOASSOCIATED VIRUS; INTRAMUSCULAR INJECTION; SUSTAINED EXPRESSION; SARCOGLYCAN COMPLEX; DELTA-SARCOGLYCAN; INTEGRATION; MICE; TRANSDUCTION; TISSUE; CELLS;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
38
Recensione:
Indirizzi per estratti:
Indirizzo: Xiao, X Univ Pittsburgh, Dept Mol Genet & Biochem, Sch Med, Room W1244,BST, Pittsburgh, PA 15261 USA Univ Pittsburgh Room W1244,BST Pittsburgh PA USA15261 A 15261 USA
Citazione:
X. Xiao et al., "Full functional rescue of a complete muscle (TA) in dystrophic hamsters byadeno-associated virus vector-directed gene therapy", J VIROLOGY, 74(3), 2000, pp. 1436-1442

Abstract

Limb girdle muscular dystrophy (LGMD) 2F is caused by mutations in the delta-sarcoglycan (SC) gene. Previously, we have shown successful application of a recombinant adeno-associated virus (AAV) vector for genetic and biochemical rescue in the Bio14.6 hamster, a homologous animal model for LGMD 2F (J. Li et al., Gene Ther, 6:74-82, 1999), In this report, we show efficientand long-term delta-SG expression accompanied by nearly complete recovery of physiological function deficits after a single-dose AAV vector injectioninto the tibialis anterior muscle of the dystrophic hamsters, AAV vector treatment led to more than 97% recovery in muscle strength for both the specific twitch forte and the specific tetanic force, when compared to the age-matched control, Vector treatment also prevented pathological muscle hypertrophy and resulted in normal muscle weight and size. Finally, vector-treated muscle showed substantial improvement of the histopathology, This is the first report of successful functional rescue of an entire muscle after AAV-mediated gene delivery. This report also demonstrates the feasibility of invivo gene therapy for LGMD patients by using AAV vectors.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 29/11/20 alle ore 10:30:27