Molecular design of functional polymers for gene therapy

In the post genomic era, massive genetic information of the human being accelerates the identification of target genes for treating various diseases. A wide range of nucleic acid therapeutics have recently emerged as powerful new drug entities for treatment of gene-related diseases and for lead validation in the drug discovery process. The absence of an efficient delivery system for genetic drugs has been identified as a major hurdle for clinical applications. Various bio-mimicking polymers have been considered as potential nonviral carriers for gene therapy due to their safety in clinical use, despite their lower gene-transduction capacity compared to virus-based gene carriers that hold risk of potential immunogenicity and endogenous virus recombination. Improving specificity toward target cells and intracellular gene-transferring efficiency will be a prerequisite for successful clinical application of polymeric gene carriers. Multifunctional polymeric gene delivery carriers should be rationally designed at the molecular level for effective human gene therapy.