Multifuctional peptides as biocompatible non-viral vectors for gene delivery

Abstract

The major barrier in gene therapy is the development of gene delivery vectors with cell-type specificity and minimal toxicity. Non-viral vectors are gaining more attention than viral vectors due to the safety consideration in clinical use. To achieve efficient delivery, non-viral vectors are modified to mimic the viral attributes such as interaction with cell surface, membrane fusion, and nuclear localization. In our studies, we constructed biocompatible peptide and polypeptide based vectors for gene delivery. The main features of these vectors are: (1) strong binding and condensation of DNA; (2) favourable positive zeta potential and nanometer-size of DNA/vector complex; (3) efficient endosomal escape of the DNA/vector complex, which is a major limiting factor in gene delivery; (4) insignificant cytotoxicity, which is a main requirement for gene therapy. Hence, these vectors can serve as a platform for developing more efficient vectors by including many different functional domains.