Molecular design of functional polymers for gene therapy

摘要

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.