| [Full Text] [PubMed URL] | |
| Journal: | Trends Biotechnol. 2000 Sep;18(9):394-9. |
| Authors: | de Grey AD. |
| Location/Contact: | Department of Genetics, University of Cambridge, Cambridge, UK CB2 3EH. ag24@gen.cam.ac.uk |
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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.Publication Types:
PMID: 10942964 [PubMed - indexed for MEDLINE] |