||NIH COVID-19 SIG Lecture Series
High throughput, combinatorial approaches have revolutionized small-molecule drug discovery. Dan Anderson will describe his work on the combinatorial development of nanoparticulate, intracellular delivery systems for RNA therapy and gene editing. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated, and screened for their ability to deliver macromolecular payloads inside of cells. These nanoformulations facilitate in vivo delivery, enabling gene suppression with small-interfering RNA, gene expression with messenger RNA, or permanent genetic editing using the CRISPR/Cas9 system. Formulations have been developed with in vivo efficacy, and show potential therapeutic applications for a range of different diseases. This lecture will focus on the application of these formulations toward controlling the immune system and in particular as vaccines for infectious disease.