The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice. (Articolo in rivista)

Type
Label
  • The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice. (Articolo in rivista) (literal)
Anno
  • 2010-01-01T00:00:00+01:00 (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#doi
  • 10.1093/hmg/ddp539 (literal)
Alternative label
  • Di Certo MG1; Corbi N2; Strimpakos G1,7; Onori A2; Luvisetto S3; Severini C1; Guglielmotti A4; Batassa EM2; Pisani C2,7; Floridi A7; Benassi B5,8; Fanciulli M5,8; Magrelli A6; Mattei E1,8; Passananti C2,8 (2010)
    The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice.
    in Human molecular genetics (Print)
    (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#autori
  • Di Certo MG1; Corbi N2; Strimpakos G1,7; Onori A2; Luvisetto S3; Severini C1; Guglielmotti A4; Batassa EM2; Pisani C2,7; Floridi A7; Benassi B5,8; Fanciulli M5,8; Magrelli A6; Mattei E1,8; Passananti C2,8 (literal)
Pagina inizio
  • 752 (literal)
Pagina fine
  • 760 (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#altreInformazioni
  • 2009 Dec 16 [Epub ahead of print] (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroVolume
  • 19 (literal)
Rivista
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroFascicolo
  • 5 (literal)
Note
  • ISI Web of Science (WOS) (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#affiliazioni
  • 1 - Istituto di Neurobiologia e Medicina Molecolare, CNR, IRCCS Fondazione S. Lucia, Via del Fosso di Fiorano 64, 00143 Rome, Italy; 2 - Istituto di Biologia e Patologia Molecolari, CNR, c/o Regina Elena Cancer Institute, Via delle Messi d'Oro 156, 00158 Rome, Italy; 3 - Istituto di Neuroscienze, CNR, Via del Fosso di Fiorano 64, 00143 Rome, Italy; 4 - Angelini Research Center, Rome, Italy; 5 - Department of Therapeutic Programs Development, Regina Elena Cancer Institute, Via E. Chianesi 53, 00144 Rome, Italy; 6 - National Centre for Rare Diseases, Istituto Superiore di Sanità, Viale Regina Elena 299, 00161 Rome, Italy; 7 - Department of Experimental Medicine, University of L'Aquila, Via Vetoio Coppito 2, 67100 L'Aquila, Italy; 8 - Rome Oncogenomic Center, Regina Elena Cancer Institute, Via E. Chianesi 53, 00144 Rome, Italy (literal)
Titolo
  • The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice. (literal)
Abstract
  • The absence of the cytoskeletal protein dystrophin results in Duchenne muscular dystrophy (DMD). The utrophin protein is the best candidate for dystrophin replacement in DMD patients. To obtain therapeutic levels of utrophin expression in dystrophic muscle, we developed an alternative strategy based on the use of artificial zinc finger transcription factors (ZF ATFs). The ZF ATF 'Jazz' was recently engineered and tested in vivo by generating a transgenic mouse specifically expressing Jazz at the muscular level. To validate the ZF ATF technology for DMD treatment we generated a second mouse model by crossing Jazz-transgenic mice with dystrophin-deficient mdx mice. Here, we show that the artificial Jazz protein restores sarcolemmal integrity and prevents the development of the dystrophic disease in mdx mice. This exclusive animal model establishes the notion that utrophin-based therapy for DMD can be efficiently developed using ZF ATF technology and candidates Jazz as a novel therapeutic molecule for DMD therapy. (literal)
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