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Titolo:
EX-VIVO GENE-TRANSFER USING ADENOVIRUS-MEDIATED FULL-LENGTH DYSTROPHIN DELIVERY TO DYSTROPHIC MUSCLES
Autore:
FLOYD SS; CLEMENS PR; ONTELL MR; KOCHANEK S; DAY CS; YANG J; HAUSCHKA SD; BALKIR L; MORGAN J; MORELAND MS; FEERO GW; EPPERLY M; HUARD J;
Indirizzi:
CHILDRENS HOSP PITTSBURGH,MUSCULOSKELETAL RES CTR,DEPT ORTHOPAED SURGPITTSBURGH PA 15261 CHILDRENS HOSP PITTSBURGH,MUSCULOSKELETAL RES CTR,DEPT ORTHOPAED SURGPITTSBURGH PA 15261 UNIV PITTSBURGH,DEPT NEUROL PITTSBURGH PA 15260 UNIV PITTSBURGH,DEPT PHYSIOL & CELL BIOL PITTSBURGH PA 15260 UNIV PITTSBURGH,DEPT HUMAN GENET PITTSBURGH PA 15260 UNIV PITTSBURGH,DEPT BIOCHEM & MOL GENET PITTSBURGH PA 15260 UNIV PITTSBURGH,DEPT RADIAT ONCOL PITTSBURGH PA 15260 BAYLOR COLL MED,DEPT MOL & HUMAN GENET HOUSTON TX 77030 UNIV WASHINGTON,DEPT BIOCHEM SEATTLE WA 98195 CHARING CROSS & WESTMINSTER MED SCH,DEPT HISTOPATHOL LONDON W6 8RF ENGLAND
Titolo Testata:
Gene therapy
fascicolo: 1, volume: 5, anno: 1998,
pagine: 19 - 30
SICI:
0969-7128(1998)5:1<19:EGUAFD>2.0.ZU;2-V
Fonte:
ISI
Lingua:
ENG
Soggetto:
DUCHENNE MUSCULAR-DYSTROPHY; HUMAN MYOBLAST TRANSPLANTATION; SKELETAL-MUSCLE; MDX MOUSE; IN-VIVO; FK506 IMMUNOSUPPRESSION; GLYCOPROTEIN COMPLEX; MINIDYSTROPHIN GENE; CELLULAR-IMMUNITY; MYOGENIC CELLS;
Keywords:
DYSTROPHIN; MDX MOUSE; ADENOVIRUS; EX VIVO APPROACH; DYSTROPHIN-ASSOCIATED PROTEINS;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Science Citation Index Expanded
Science Citation Index Expanded
Science Citation Index Expanded
Citazioni:
77
Recensione:
Indirizzi per estratti:
Citazione:
S.S. Floyd et al., "EX-VIVO GENE-TRANSFER USING ADENOVIRUS-MEDIATED FULL-LENGTH DYSTROPHIN DELIVERY TO DYSTROPHIC MUSCLES", Gene therapy, 5(1), 1998, pp. 19-30

Abstract

Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle disease characterized by a lack of dystrophin expression. Myoblast transplantation and gene therapy have the potential of restoring dystrophin,thus decreasing the muscle weakness associated with the disease. In this study we present data on the myoblast mediated ex vivo gene transfer of full-length dystrophin to mdx (dystrophin deficient) mouse muscle as a model for autologous myoblast transfer. Both isogenic primary mdx myoblasts and an immortalized mdx cell line were transduced with anadenoviral vector that has all viral coding sequences deleted and encodes beta-galactosidase and full-length dystrophin. Subsequently, these transduced myoblasts were injected into dystrophic max muscle, wherethe injected cells restored dystrophin, as well as dystrophin-associated proteins. A greater amount of dystrophin replacement occurred in mdx muscle following transplantation of mdx myoblasts isolated from a transgenic mouse overexpressing dystrophin suggesting that engineering autologous myoblasts to express high amounts of dystrophin might be beneficial. The ex vivo approach possesses attributes that make it useful for gene transfer to skeletal muscle including: (1) creating a reservoir of myoblasts capable of regenerating and restoring dystrophin to dystrophic muscle; and (2) achieving a higher level of gene transfer to dystrophic muscle compared with adenovirus-mediated direct gene delivery. However, as observed in direct gene transfer studies, the ex vivo approach also triggers a cellular immune response which limits the duration of transgene expression.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 08/07/20 alle ore 06:37:44