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Web Date: November 22, 2016

A step toward mitochondrial gene therapy

A self-assembling, peptide-based gene delivery system might one day correct mitochondrial DNA mutations that underlie disease
Department: Science & Technology
Keywords: biochemistry, mitochondria, mitochondrial disease, gene therapy, DNA delivery

Mutations in mitochondrial DNA underlie many diseases, including cancer, diabetes, heart disease, and age-related neurodegenerative disorders. But a safe, reliable method to deliver DNA to mitochondria and correct these mutations doesn’t currently exist.

Now, a team led by biochemistry professor Keiji Numata and postdoctoral researcher Jo-Ann Chuah of Japan’s RIKEN research institute have developed a peptide-based system that delivers a functional gene to human cells without toxic effects. The system could pave . . .

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Chemical & Engineering News
ISSN 0009-2347
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