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Titolo:
Prospects of gene therapy for muscular dystrophy
Autore:
Lochmuller, H;
Indirizzi:
Univ Munich, Genzentrum, D-81377 Munich, Germany Univ Munich Munich Germany D-81377 , Genzentrum, D-81377 Munich, Germany Univ Munich, Friedrich Baur Inst, D-81377 Munich, Germany Univ Munich Munich Germany D-81377 ch Baur Inst, D-81377 Munich, Germany
Titolo Testata:
NERVENHEILKUNDE
fascicolo: 1, volume: 18, anno: 1999,
pagine: 14 - 16
SICI:
0722-1541(199901)18:1<14:POGTFM>2.0.ZU;2-U
Fonte:
ISI
Lingua:
GER
Soggetto:
ADENOVIRUS-MEDIATED TRANSFER; MDX MICE; IN-VIVO; MINIDYSTROPHIN GENE; CYSTIC-FIBROSIS; SKELETAL-MUSCLE; EXPRESSION; IMMUNOSUPPRESSION; RECOMBINANTS; EFFICIENCY;
Keywords:
Duchenne's muscular dystrophy; gene therapy; adenoviral gene transfer; dystrophin;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
21
Recensione:
Indirizzi per estratti:
Indirizzo: Lochmuller, H Univ Munich, Genzentrum, Feodor Lynen Str 25, D-81377 Munich, Germany Univ Munich Feodor Lynen Str 25 Munich Germany D-81377 many
Citazione:
H. Lochmuller, "Prospects of gene therapy for muscular dystrophy", NERVENHEILK, 18(1), 1999, pp. 14-16

Abstract

Replication-deficient adenovirus vectors (AdV) have been successfully usedto transfer a truncated human dystrophin cDNA to skeletal muscle of dystrophin-deficient mdx mice. However, a humoral and cellular immune response ofthe host against antigens of viral and transgene origin leads to a declineof dystrophin expression over a 2 month period. Immunosuppression significantly prolongs transgene expression. Animal models may help to solve the open questions pertaining to dystrophin gene transfer such as systemic delivery and improvement of muscle function, before human trials for gene replacement therapy in DMD may be considered.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 08/04/20 alle ore 09:12:51