Materials for non-viral gene delivery

Novel therapeutic strategies can be envisioned based on altering the expres sion level of target genes involved in cellular processes and disease progr ession; however, our ability to efficiently manipulate gene expression is l imited. Non-viral-based gene therapy provides a relatively safe approach to increase or decrease the expression of a specific gene using DNA or antise nse sequences; however, synthetic systems are required to direct plasmids a nd oligonucleotides to a specific tissue and to enhance cellular uptake and intracellular trafficking. Numerous materials are being developed that int eract with DNA to enhance its properties (e.g. stability, charge density) a nd thus direct its biodistribution and facilitate cellular interactions. Th e development of synthetic delivery systems to manipulate gene expression e fficiently is a powerful tool that will ultimately lead to novel therapeuti c strategies for the treatment of numerous disorders.