Ryan (Hung-Hsun) Lu – UMass Trainee 2023-2025
My project is focused on the development of photocrosslinked RNA delivery system to overcome extracellular and intracellular barriers in the systemic delivery. Gene therapy has emerged as a novel therapeutic strategy and is seen as a precision medicine due to its sequence-specific manner, as witnessed by the increasing numbers of FDA-approved RNA therapeutics. For systemic administration, siRNA and mRNA therapeutics rely
on delivery vehicles that protect the RNA from enzymatic degradation while still allowing cellular uptake and release into cytoplasm. Current delivery vehicles in the market are limited to adeno-associated virus (AAV) and lipid nanoparticles. Severe side effects such as acute liver and kidney failure were found in FDA-approval LUXTURNA® and ZOLGENSMA® clinical trials.