Recombinant protein polymer vectors for systemic gene delivery

摘要

The present invention relates to genetically engineered non-viral vectors for delivering a nucleic acid such as a therapeutic gene to a target cell. The vectors are suitable for systemic administration to an animal. In the simplest embodiment the non-viral vector is a nucleic acid-binding protein-based polymer (NABP) having at least one tandem repeat of a genetically engineered cationic amino acid-containing monomer (CAACM) containing lysine, arginine or a combination thereof, which confers on the NABP the ability to bind a nucleic acid that is intended for delivery to a target cell. Because the NABP is genetically engineered and transcribed from a single gene, its structure and function can be precisely controlled. The vectors optionally have additional functionalities including endosome disrupting moieties, targeting ligands and subcellular localization sequences.