ABSTRACT
Lead polymers have efficacy comparable to adenovirus for gene delivery in vitro and, with cytotoxic DNA payloads, can induce tumor regression in vivo. The expression of missing genes can be turned on by delivering exogenous DNA, and the expression of undesirable genes can be turned off through silencing siRNA or antisense. Viruses have evolved to be highly efficient at delivering genetic cargo to host cells and researchers have exploited this fact to modify viruses to deliver therapeutic genes of interest. Alternative viral strategies include adeno-associated virus and herpes simplex virus, although the approaches have significant safety, manufacturing, and cargo capacity concerns as well. Adeno-associated virus has also shown an insertional mutagenesis risk in mice. The chapter describes combinatorial libraries of biomaterials that are useful for genetic nanomedicine. High-throughput synthesis and screening methods were discussed that allow for parallel nanoparticle fabrication and cell-based testing.
