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• W2170639503 abstract "Mitochondrial DNA (mtDNA) mutations underlie many rare diseases and might also contribute to human ageing. Gene therapy is a tempting future possibility for intervening in mitochondriopathies. Expression of the 13 mtDNA-encoded proteins from nuclear transgenes (allotopic expression) might be the most effective gene-therapy strategy. Its only confirmed difficulty is the extreme hydrophobicity of these proteins, which prevents their import into mitochondria from the cytosol. Inteins (self-splicing 'protein introns') might offer a solution to this problem: their insertion into such transgenes could greatly reduce the encoded proteins' hydrophobicity, enabling import, with post-import excision restoring the natural amino acid sequence." @default.
• W2170639503 created "2016-06-24" @default.
• W2170639503 creator A5079224031 @default.
• W2170639503 date "2000-09-01" @default.
• W2170639503 modified "2023-10-02" @default.
• W2170639503 title "Mitochondrial gene therapy: an arena for the biomedical use of inteins" @default.
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• W2170639503 doi "https://doi.org/10.1016/s0167-7799(00)01476-1" @default.
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