期刊
NUCLEIC ACIDS RESEARCH
卷 41, 期 1, 页码 418-433出版社
OXFORD UNIV PRESS
DOI: 10.1093/nar/gks965
关键词
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资金
- CNRS (Centre National de Recherche Scientifique)
- University of Strasbourg
- AFM (Association Francaise contre les Myopathies)
- ANR (Agence Nationale de la Recherche)
- FRM (Fondation pour la Recherche Medicale)
- ARCUS/Suprachem collaboration program
- LIA collaboration program (ARNmitocure)
- National Program 'Investissement d'Avenir' (Labex MitoCross)
- FRM
- AFM
Mitochondrial mutations, an important cause of incurable human neuromuscular diseases, are mostly heteroplasmic: mutated mitochondrial DNA is present in cells simultaneously with wild-type genomes, the pathogenic threshold being generally > 70% of mutant mtDNA. We studied whether heteroplasmy level could be decreased by specifically designed oligoribonucleotides, targeted into mitochondria by the pathway delivering RNA molecules in vivo. Using mitochondrially imported RNAs as vectors, we demonstrated that oligoribonucleotides complementary to mutant mtDNA region can specifically reduce the proportion of mtDNA bearing a large deletion associated with the Kearns Sayre Syndrome in cultured transmitochondrial cybrid cells. These findings may be relevant to developing of a new tool for therapy of mtDNA associated diseases.
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