About This Event
ZOOM LINKMeeting ID: 937 4316 0035Passcode: 411385 Genetic medicines have the potential to treat various diseases, but require development of controlled delivery vehicles to transfect specific cell types in specific organs to achieve therapeutic effects. Moreover, certain ailments including inflammatory diseases and cancer would benefit from control over intracellular and extracellular localization of therapeutic proteins. In this presentation, I will discuss the development of selective organ targeting (SORT) lipid nanoparticles (LNPs) that enable high levels of genome editing in the liver, lungs, spleen, tumors, or bone marrow. I will also discuss signal peptide engineered nucleic acid design (SEND) mRNAs that encode for etanercept/TNF-α inhibitor antibodies and demonstrate therapeutic efficacy in a psoriasis model by reducing hyperkeratosis and inflammation. The modular nature of SEND enables intracellular and extracellular localization control of various functional proteins for diverse therapeutic applications. Cumulatively, these findings introduce strategies to control how delivery nanoparticles navigate to where they need to go, control how delivery nanoparticles release nucleic acids inside of cells, and control how translated proteins navigate to where they need to go for ultimate therapeutic benefit.
