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

doi:10.1159/000064726

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Article

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

Journal of biomedical science - 1 Jan 2002

Tanaka Masashi, Borgeld Harm-Jan, Zhang Jin, Muramatsu Shin-ichi, Gong Jian-Sheng, Yoneda Makoto, Maruyama Wakako, Naoi Makoto, Ibi Tohru, Sahashi Ko, Shamoto Masayo, Fuku Noriyuki, Kurata Miyuki, Yamada Yoshiji, Nishizawa Kumi, Akao Yukihiro, Ohishi Nobuko, Miyabayashi Shigeaki, Umemoto Hiraku, Muramatsu Tatsuo, Furukawa Koichi, Kikuchi Akihiko, Nakano Imaharu, Ozawa Keiya, Yagi Kunio

Abstract excerpt

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...

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PMID: 12372991
DOI: 10.1159/000064726

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

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