SITE SPECIFIC DELIVERY OF NUCLEIC ACIDS BY COMBINING TARGETING LIGANDS WITH ENDOSOMOLYTIC COMPONENTS

摘要

PROBLEM TO BE SOLVED: To provide compositions for site-specific delivery of nucleic acids in treatment of cancer, viral disease etc.SOLUTION: There is provided a modular composition containing a nucleic acid, an endosomolytic component, and a targeting ligand, as represented by the structure in the figure as a representative example. [E is the endosomolytic component; L is the targeting ligand; x are each independently 0 or 1; y are each independently an integer from 1 to 6; z are each independently an integer from 0 to 6; and A is a linking moiety having pyrrolidine and phosphate ester structure.]