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Titolo:
OVEREXPRESSION OF DYSTROPHIN IN TRANSGENIC MDX MICE ELIMINATES DYSTROPHIC SYMPTOMS WITHOUT TOXICITY
Autore:
COX GA; COLE NM; MATSUMURA K; PHELPS SF; HAUSCHKA SD; CAMPBELL KP; FAULKNER JA; 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 MICHIGAN,SCH MED,INST GERONTOL ANN ARBOR MI 48109 UNIV MICHIGAN,SCH MED,CTR HUMAN GENOME ANN ARBOR MI 48109 UNIV IOWA,COLL MED,HOWARD HUGHES MED INST IOWA CITY IA 52242 UNIV IOWA,COLL MED,DEPT PHYSIOL & BIOPHYS IOWA CITY IA 52242 UNIV WASHINGTON,DEPT BIOCHEM SEATTLE WA 98195
Titolo Testata:
Nature
fascicolo: 6439, volume: 364, anno: 1993,
pagine: 725 - 729
SICI:
0028-0836(1993)364:6439<725:OODITM>2.0.ZU;2-W
Fonte:
ISI
Lingua:
ENG
Soggetto:
DUCHENNE MUSCULAR-DYSTROPHY; SKELETAL-MUSCLE; EXPRESSION; MOUSE; GLYCOPROTEIN; PROTEIN; DEFICIENCY; MEMBRANE; COMPLEX;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Citazioni:
27
Recensione:
Indirizzi per estratti:
Citazione:
G.A. Cox et al., "OVEREXPRESSION OF DYSTROPHIN IN TRANSGENIC MDX MICE ELIMINATES DYSTROPHIC SYMPTOMS WITHOUT TOXICITY", Nature, 364(6439), 1993, pp. 725-729

Abstract

DUCHENNE and Becker muscular dystrophy (DMD and BMD) are X-linked recessive diseases caused by defective expression of dystrophin1,2. The mdx mouse, an animal model for DMD, has a mutation that eliminates expression of the 427K muscle and brain isoforms of dystrophin1,3,4. Although these animals do not display overt muscle weakness or impaired movement, the diaphragm muscle of the mdx mouse is severely affected and shows progressive myofibre degeneration and fibrosis which closely resembles the human disease5,6. Here we explore the feasibility of gene therapy for DMD by examining the potential of a full-length dystrophin transgene to correct dystrophic symptoms in mdx mice. We find that expression of dystrophin in muscles of transgenic mdx mice eliminates themorphological and immunohistological symptoms of muscular dystrophy. In addition, overexpression of dystrophin prevents the development of the abnormal mechanical properties associated with dystrophic muscle without causing deleterious side effects. Our results provide functional evidence for the feasibility of gene therapy for DMD.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 25/09/20 alle ore 00:33:20