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Titolo:
ADENOVIRUS-MEDIATED DYSTROPHIN MINIGENE TRANSFER IMPROVES MUSCLE STRENGTH IN ADULT DYSTROPHIC (MDX) MICE
Autore:
YANG L; LOCHMULLER H; LUO J; MASSIE B; NALBANTOGLU J; KARPATI G; PETROF BJ;
Indirizzi:
ROYAL VICTORIA HOSP,DIV RESP,ROOM L408,687 PINE AVE W QUEBEC CITY PQ H3A 1A1 CANADA ROYAL VICTORIA HOSP,DIV RESP QUEBEC CITY PQ H3A 1A1 CANADA MCGILL UNIV,MEAKINS CHRISTIE LABS MONTREAL PQ H3A 2T5 CANADA MCGILL UNIV,MONTREAL NEUROL INST,NEUROMUSCULAR RES GRP MONTREAL PQ H3A 2T5 CANADA GENZENTRUM MUNICH GERMANY FRIEDRICH BAUR INST MUNICH GERMANY NATL RES COUNCIL CANADA,BIOTECHNOL RES INST MONTREAL PQ H4P 2R2 CANADA
Titolo Testata:
Gene therapy
fascicolo: 3, volume: 5, anno: 1998,
pagine: 369 - 379
SICI:
0969-7128(1998)5:3<369:ADMTIM>2.0.ZU;2-1
Fonte:
ISI
Lingua:
ENG
Soggetto:
DUCHENNE MUSCULAR-DYSTROPHY; VIVO GENE-TRANSFER; FULL-LENGTH; MINIDYSTROPHIN GENE; MOUSE DIAPHRAGM; SKELETAL-MUSCLE; THERAPY; EXPRESSION; FIBERS; RECOMBINANTS;
Keywords:
DUCHENNE MUSCULAR DYSTROPHY; MDX DIAPHRAGM; VIRAL TOXICITY; IMMUNOSUPPRESSION; MUSCLE FUNCTION;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Science Citation Index Expanded
Science Citation Index Expanded
Science Citation Index Expanded
Citazioni:
39
Recensione:
Indirizzi per estratti:
Citazione:
L. Yang et al., "ADENOVIRUS-MEDIATED DYSTROPHIN MINIGENE TRANSFER IMPROVES MUSCLE STRENGTH IN ADULT DYSTROPHIC (MDX) MICE", Gene therapy, 5(3), 1998, pp. 369-379

Abstract

Duchenne muscular dystrophy (DMD) and murine X-linked muscular dystrophy (mdx) are both due to absence of the subsarcolemmal protein dystrophin. Recombinant adenovirus vectors (AdV) are considered a promising means for delivering a functional dystrophin gene to muscle. However, the usefulness of AdV for this purpose is limited by vector toxicity as well as immune-mediated elimination of infected fibers. In addition,studies to date of AdV-mediated dystrophin gene transfer have either failed to examine effects on muscle strength or been performed in immunologically immature neonatal animals with little baseline abnormalityof force-generating capacity. In the present study, AdV-mediated dystrophin gene transfer was performed in adult mdx mice with pre-existentdystrophic pathology and muscle weakness. The main findings are as follows: (1) acute myofiber toxicity and gene transfer efficiency are both AdV dose-dependent, such that the therapeutic margin of safety is fairly narrow; (2) immunosuppressive therapy (FK506) prevents immune-mediated elimination of dystrophin-positive fibers but not the dose-dependent toxic effects; (3) at the optimal vector dosage and with effective immunosuppression, AdV-mediated dystrophin minigene transfer is capable of alleviating the loss of force-generating capacity as well as histopathological evidence of disease progression normally seen in adult mdx muscles over a 2-month period. These findings have important implications for the eventual application of AdV-mediated dystrophin genetransfer in DMD patients.

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Documento generato il 04/04/20 alle ore 14:59:45