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CNR: Alamanacco della Scienza


N. 2 - 24 mar 2010
ISSN 2037-4801

International info   a cura di Cecilia Migali


Cnr study on artificial genes as therapeutic molecules

Duchenne Muscular Dystrophy (Dmd) is a severe muscle degenerative disease, due to absence of dystrophin. There is currently no effective treatment for Dmd. The up-regulation of the dystrophin related gene utrophin, to compensate for dystrophin absence, is one of the most promising strategy for DMD treatment.

To obtain therapeutic levels of utrophin gene expression in dystrophic muscle, two Cnr research groups developed an innovative strategy based on the use of artificial zinc finger transcription factors (ZF ATFs) (Corbi et al. Gene Therapy 2000, Onori et al. Biochem Cell Biol 2007). In particular, Claudio Passananti and Elisabetta Mattei teams (Institute of Molecular Biology and Pathology and Institute of Neurobiology and Molecular Medicine, Rome) generated a transgenic mouse expressing the artificial mini-gene, called Jazz, codifying for a transcription factor able to up-regulate utrophin expression at the muscular level (Mattei et al. PloS One, 2007).

The synthetic Jazz transcription factor is well tolerated in vivo and it is safe for the health of the transgenic animal throughout its span life. It is demonstrated that the cross-breading of Jazz-mouse with "dystrophic" mouse model (mdx) displays a strong regression/prevention of the Dmd pathology (Di Certo et al. Hum Mol Genet. 2009). Therefore, Jazz-based ZF ATFs are good candidates as novel therapeutic molecules for Dmd treatment.The results were patented by the Cnr.

Moreover, with the fundamental support of the Department of Life Sciences (Dsv), the Cnr has recently signed a term sheet for cooperation and license with the Israeli ILIT Bio-Ventures Company.

Further information

Fonte: Elisabetta Mattei, Istituto di biologia e patologia molecolari, Roma, email elisabetta.mattei@inmm.cnr.it

Fonte: Claudio Passananti, Istituto di biologia e patologie molecolari del Cnr presso l'ospedale 'Regina Elena' di Roma, tel. 06/52662573, email claudio.passananti@ibpm.cnr.it