Xiaoshu Pan, PhD
Leveraging Extracellular Vesicles for Gene Therapy in Hearing Restoration
Abstract
Extracellular vesicles(EVs) present a remarkable potential for precise, safe, and effective therapeutic delivery within the inner ear system. However, the challenge of efficiently loading therapeutic cargoes into EVs persists, impeding clinical translation. I have introduced an innovative continuous-flow droplet microfluidic electroporation system. This advancement enhanced throughput and CRISPR genome editing complex transfection into EVs, demonstrated by heightened inner ear cell transfection compared to lipid-like nanoparticles. In this proposal, I will integrate selection tags for cargo enrichment and electroporation enhancers like gold nanoparticles and pH-responsive transmembrane insertion peptides, further optimizing EV-based gene therapy for inner ear applications. Ultimately, spatial imaging and differential multi-omics analysis of EVs will enhance the molecular comprehension of cargo-loaded EVs that may guide the clinical translation of EV-based gene therapy.
Pan and her labmates
I am truly grateful for the support of PhRMA Foundation to advance our technology for developing advanced gene therapy. It's a recognition of the potential impact my work can have on gene delivery. I am excited about the possibilities it brings for my future endeavors in advancing gene therapy.
