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 J Borgeld and Jin Zhang and Shin-ichi Muramatsu and Jian-Sheng Gong and Makoto Yoneda and Wakako Maruyama and Makoto Naoi and Tohru Ibi and Kazuki Sahashi and Masayo Shamoto and Noriyuki Fuku and Miyuki Kurata and Yoshiji Yamada and Kumi Nishizawa and Yukihiro Akao and Nobuko Ohishi and Shigeaki Miyabayashi and Hiraku Umemoto and Tatsuo Muramatsu and Koichi Furukawa and Akihiko Kikuchi and Imaharu Nakano and Keiya Ozawa and Kunio 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… CONTINUE READING
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