Transfection of mitochondria: strategy towards a gene therapy of mitochondrial DNA diseases

@article{Seibel1995TransfectionOM,
  title={Transfection of mitochondria: strategy towards a gene therapy of mitochondrial DNA diseases},
  author={Peter Seibel and J{\"o}rg Trappe and Gaetano Villani and Thomas Klopstock and Sergio Papa and Heinz Reichmann},
  journal={Nucleic acids research},
  year={1995},
  volume={23 1},
  pages={10-7}
}
Successes in classical gene therapies have been achieved by placing a corrected copy of a defective nuclear gene in cells. A similar gene replacement approach for a mutant mitochondrial genome is invariably linked to the use of a yet unavailable mitochondrial transfection vector. Here we show that DNA coupled covalently to a short mitochondrial leader peptide (chimera) can enter mitochondria via the protein import pathway, opening a new way for gene-, antisense-RNA- or antisense-DNA-delivery in… CONTINUE READING
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