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Eine gentherapeutische Strategie zur Behandlung von Energiestoffwechsel‐Erkrankungen
Author(s) -
Seibel Peter,
Trappe Jörg
Publication year - 1996
Publication title -
chemie in unserer zeit
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.217
H-Index - 24
eISSN - 1521-3781
pISSN - 0009-2851
DOI - 10.1002/ciuz.19960300504
Subject(s) - mitochondrial dna , nucleic acid , mitochondrion , somatic cell , transfection , gene , genetic enhancement , biology , organelle , genome , inner mitochondrial membrane , chemistry , dna , microbiology and biotechnology , biochemistry
Genetic variations of the mitochondrial genome lead to severe neuromuscular diseases in man. A treatment of these utilizing a somatic gene therapy approach is invariably linked to a mitochondrial transfection system. A novel technique of targeting nucleic acids to mitochondria has been developed that takes advantage of the protein import pathway. The system is based on chimerical molecules that are composed of a DNA and a protein moiety, harboring the information for mitochondrial targeting. Upon recognition of these molecules by a receptor on the outer mitochondrial membrane, the molecules are able to cross the membrane system and are released into the matrix of the organelle. The further development of this technique will give to rise to strategies for the treatment of mitochondrial DNA diseases by a somatic gene therapy approach.