Nucleic acid therapeutics can be drug molecules with high programmability, minimal off-target effects, and the capability to address “undruggable” targets for diseases. In addition, each time a new drug is needed, one needs to only change the sequence as opposed to finding an entirely new structure. One nucleic acid type...
Cardiovascular disease is a leading cause of morbidity and mortality worldwide. While many therapies have been used to treat vascular disease symptomatically, gene therapy offers a means of treating the molecular dysfunctions driving disease pathogenesis. Unfortunately, gene therapy has achieved only modest therapeutic success in clinical trials due to a...
Although non-viral gene therapy has great potential for use in the lung, several problems including inefficient gene transfer and expression and the relative lack of cell-specific targeting have limited its applications. The two approaches that have been used to target genes to desired cell types are through specific ligand-receptor interactions...