Publications

Found 7 results
Filters: Keyword is Genetic Therapy  [Clear All Filters]
2014
Bottani E, Giordano C, Civiletto G, Di Meo I, Auricchio A, Ciusani E, Marchet S, Lamperti C, d'Amati G, Viscomi C & Zeviani M (2014) AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failure. Mol Ther 22, 10-7
Torres-Torronteras J, Viscomi C, Cabrera-Pérez R, Cámara Y, Di Meo I, Barquinero J, Auricchio A, Pizzorno G, Hirano M, Zeviani M & Martí R (2014) Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE. Mol Ther 22, 901-7
Gammage PA, Rorbach J, Vincent AI, Rebar EJ & Minczuk MA (2014) Mitochondrially targeted ZFNs for selective degradation of pathogenic mitochondrial genomes bearing large-scale deletions or point mutations. EMBO Mol Med 6, 458-66
2012
Di Meo I, Auricchio A, Lamperti C, Burlina A, Viscomi C & Zeviani M (2012) Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy. EMBO Mol Med 4, 1008-14
2003
Zeviani M & Carelli V (2003) Mitochondrial disorders. Curr Opin Neurol 16, 585-94
2001
Muratovska A, Lightowlers RN, Taylor RW, Wilce JA & Murphy MP (2001) Targeting large molecules to mitochondria. Adv Drug Deliv Rev 49, 189-98