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Titolo:
Gene therapy for muscular dystrophies - Current status and future prospects
Autore:
Takeda, S; Miyagoe-Suzuki, Y;
Indirizzi:
Natl Ctr Neurol & Psychiat, Dept Mol Therapy, Natl Inst Neurosci, Kodaira,Tokyo 1878502, Japan Natl Ctr Neurol & Psychiat Kodaira Tokyo Japan 1878502 kyo 1878502, Japan
Titolo Testata:
BIODRUGS
fascicolo: 10, volume: 15, anno: 2001,
pagine: 635 - 644
SICI:
1173-8804(2001)15:10<635:GTFMD->2.0.ZU;2-C
Fonte:
ISI
Lingua:
ENG
Soggetto:
MDX MOUSE MUSCLE; ADENOVIRUS-MEDIATED TRANSFER; LENGTH HUMAN DYSTROPHIN; SKELETAL-MUSCLE; IN-VIVO; FULL-LENGTH; RNA/DNA OLIGONUCLEOTIDES; ADENOASSOCIATED VIRUS; SARCOGLYCAN COMPLEX; BETA-GALACTOSIDASE;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
50
Recensione:
Indirizzi per estratti:
Indirizzo: Takeda, S Natl Ctr Neurol & Psychiat, Dept Mol Therapy, Natl Inst Neurosci, 4-1 Ogawa Higashi, Kodaira, Tokyo 1878502, Japan Natl Ctr Neurol & Psychiat 4-1 Ogawa Higashi Kodaira Tokyo Japan 1878502
Citazione:
S. Takeda e Y. Miyagoe-Suzuki, "Gene therapy for muscular dystrophies - Current status and future prospects", BIODRUGS, 15(10), 2001, pp. 635-644

Abstract

Since the identification in 1987 of the gene for Duchenne muscular dystrophy (DMD), research on the molecular pathogenesis of muscular dystrophy has progressed extensively. In particular, discovery of the DMD gene product, dystrophin, led to the identification of dystrophin-associated proteins and,subsequently, the recognition of other types of muscular dystrophy caused by the defects in each of the sarcoglycan genes. On the other hand, effective therapy for DMD has not yet been established. Some of the viral vectors,such as adenoassociated virus vectors or lentiviral vector, have been proven to enable the longterm expression of the exogenous gene without overt host immune reactions. However, dystrophin cDNAs are too large (14kb) to be accommodated in these viral vectors. To solve this problem, we and other research groups succeeded in truncating full-length dystrophin cDNA to small dystrophin cDNA (4 to 5kb), the products of which protect dystrophin-deficient mdx muscle from contraction-induced membrane damage when introduced by viral vectors or as a transgene into mdx mice. The usefulness of these truncated dystrophin cDNAs should be confirmed using other animal models such asdystrophic dogs. To develop successful treatment of DMD, the authors believe that several different approaches should be used, such as cell transfer therapy, drug design to up-regulate utrophin, or a strategy to repair the mutation in vivo.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 28/11/20 alle ore 17:57:42