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Gene therapy of mitochondrial diseases using human cytoplasts

Gene Therapy volume 4, pages 6–10 (1997)Cite this article

Abstract

Mitochondrial (mt) DNA is highly susceptible to mutation. A novel approach using cytoplasts is essential for gene therapy of the diseases caused by these mutations, because of their non-Mendelian genetics. In this method, mtDNA is transferred into the mutant cells by forming cybrids and can complement the defect correctly and safely.

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Authors and Affiliations

  1. Department of Biochemistry, Jichi Medical School, Tochigi, 329–04, Japan

    Y Kagawa

  2. Institute of Biological Science, University of Tsukuba, Ibaraki, 305, Japan

    J-I Hayashi

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  1. Y Kagawa
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  2. J-I Hayashi
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Kagawa, Y., Hayashi, JI. Gene therapy of mitochondrial diseases using human cytoplasts. Gene Ther 4, 6–10 (1997). https://doi.org/10.1038/sj.gt.3300347

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  • DOI: https://doi.org/10.1038/sj.gt.3300347

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