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Titolo:
IMPROVED ADENOVIRAL VECTORS FOR GENE-THERAPY OF DUCHENNE MUSCULAR-DYSTROPHY
Autore:
HAUSER MA; AMALFITANO A; KUMARSINGH R; HAUSCHKA SD; CHAMBERLAIN JS;
Indirizzi:
UNIV MICHIGAN,SCH MED,DEPT HUMAN GENET ANN ARBOR MI 48109 UNIV MICHIGAN,SCH MED,DEPT HUMAN GENET ANN ARBOR MI 48109 UNIV WASHINGTON,DEPT BIOCHEM SEATTLE WA 98195
Titolo Testata:
Neuromuscular disorders
fascicolo: 5, volume: 7, anno: 1997,
pagine: 277 - 283
SICI:
0960-8966(1997)7:5<277:IAVFGO>2.0.ZU;2-E
Fonte:
ISI
Lingua:
ENG
Soggetto:
CREATINE-KINASE GENE; TRANSGENIC MDX MICE; MOUSE MYOBLASTS; CARDIAC-MUSCLE; EXPRESSION; SKELETAL; ELEMENTS; MUTATION; ENHANCER;
Keywords:
MDX MICE; ADENOVIRUS; GENE THERAPY; DYSTROPHIN; ENCAPSIDATED MINICHROMOSOME; CREATINE KINASE; DUCHENNE MUSCULAR DYSTROPHY;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Science Citation Index Expanded
Citazioni:
26
Recensione:
Indirizzi per estratti:
Citazione:
M.A. Hauser et al., "IMPROVED ADENOVIRAL VECTORS FOR GENE-THERAPY OF DUCHENNE MUSCULAR-DYSTROPHY", Neuromuscular disorders, 7(5), 1997, pp. 277-283

Abstract

We have been exploring the feasibility of gene therapy for Duchenne muscular dystrophy by characterizing parameters important for the design of therapeutic protocols. These studies have used transgenic mice toanalyze expression patterns of multiple dystrophin vectors, and have been accompanied by the development of viral vectors for gene transferto dystrophic mdx mouse muscle. Analysis of transgenic mdx mice indicates that greater than 50% of the fibers in a muscle group must express dystrophin to prevent development of a significant dystrophy, and that low-level expression of truncated dystrophins can function Very well. These results suggest that gene therapy of DMD will require methodsto transduce the majority of fibers in critical muscle groups with vectors that express moderate levels of dystrophin proteins. Strategies for the development of viral vectors able to deliver dystrophin genes to muscle include the use of muscle specific regulatory sequences coupled with deletion of viral gene sequences to limit virus-induced immune rejection of transduced tissues. These strategies should enable production of adenoviral vectors expressing full-length dystrophin proteins in muscle. (C) 1997 Elsevier Science B.V.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 29/09/20 alle ore 19:38:45