New chimaeric peptide nucleic acid construct

摘要

In order to appropriately introduce genetic material into cell organelles, the corresponding nucleic acid is bound to a cell-specific, compartment-specific or membrane-specific peptide. The nucleic acid is directed by the peptide to the aimed compartment through the natural protein transport paths and is transported through the membrane (the membrane system). For nucleic acids to unfold their replication and transcription activity in cells and cell organelles and to be projected into the cell through the protein import route, they must on the one hand have all required genetic elements and on the other hand must not exceed the pore import capacity. These two requirements caused the development of a linear-cyclic plasmid system which contains all required genetic elements, may be absorbed through the protein import route thanks to its pseudo-linear structure (cyclic DNA ends) and may finally build cyclic replication intermediates. By combining in any desired manner nucleic acids and compartment-specific protein sequences, this process allows nucleic acids to be appropriately introduced into cells and cell organelles. This system is thus suitable both for mutagenesis and in the molecular therapy of genetic diseases.