Article
Transfection of mitochondria: strategy towards a gene therapy of mitochondrial DNA diseases.
Nucleic acids research - 11 Jan 1995
Seibel P, Trappe J, Villani G, Klopstock T, Papa S, Reichmann H
Abstract excerpt
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...
Topics
- Amino Acid Sequence
- Animals
- Base Sequence
- Biological Transport, Active
- DNA, Mitochondrial
- Genetic Therapy
- In Vitro Techniques
- Mitochondria, Liver
- Molecular Sequence Data
- Mutation
- Ornithine Carbamoyltransferase