Gene therapy for mitochondrial disease by delivering restriction endonuclease SmaI into mitochondria.

@article{Tanaka2002GeneTF,
  title={Gene therapy for mitochondrial disease by delivering restriction endonuclease SmaI into mitochondria.},
  author={Masashi Tanaka and H. Borgeld and Jin Zhang and S. Muramatsu and J. Gong and M. Yoneda and W. Maruyama and M. Naoi and T. Ibi and K. Sahashi and M. Shamoto and N. Fuku and M. Kurata and Yoshiji Yamada and K. Nishizawa and Y. Akao and N. Ohishi and S. Miyabayashi and H. Umemoto and T. Muramatsu and Koichiro Furukawa and A. Kikuchi and I. Nakano and K. Ozawa and K. Yagi},
  journal={Journal of biomedical science},
  year={2002},
  volume={9 6 Pt 1},
  pages={
          534-41
        }
}
The restriction endonuclease SmaI has been used for the diagnosis of neurogenic muscle weakness, ataxia and retinitis pigmentosa disease or Leigh's disease, caused by the Mt8993T-->G mutation which results in a Leu156Arg replacement that blocks proton translocation activity of subunit a of F(0)F(1)-ATPase. Our ultimate goal is to apply SmaI to gene therapy for this disease, because the mutant mitochondrial DNA (mtDNA) coexists with the wild-type mtDNA (heteroplasmy), and because only the mutant… Expand
Current strategies towards therapeutic manipulation of mtDNA heteroplasmy.
Mitochondrial DNA heteroplasmy in disease and targeted nuclease‐based therapeutic approaches
Mitochondrial Pathogenesis
Modifying the Mitochondrial Genome.
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