Nanoparticle delivery of RNA-based therapeutics to alter the vocal fold tissue response to injury

摘要

Objective(s)Our laboratory and others hypothesize Smad3 is a principle mediator of the fibrotic phenotype in the vocal folds (VF), and we further posit that alteration of Smad3 expression through short interfering (si)RNA holds therapeutic promise, yet delivery remains challenging. To address this issue, we employed a novel synthetic oligomer, >lipitoid, complexed with siRNA to improve stability and cellular uptake with the goal of increased efficiency of RNA-based therapeutics.