Xiaoshu Pan, PhD
Leveraging Nanoparticles to Deliver Gene Therapy for Hearing Restoration
Summary
Hearing loss impacts millions worldwide, with half of these cases linked to genetic factors. Currently, there are no biological treatments available for people with deafness. Our project focuses on engineering natural extracellular vesicles (EVs) for delivering genetic medicine. EVs are tiny carriers that transport molecular “messages” in our body. We developed a scalable and effective method to mix and load drugs into EVs. We are using gold nanoparticles, which have high electrical conductivity, to enhance electrical stimulation applied to the EV membrane, making it more permeable so the genetic medicine can move into the EVs. To track these drug-loaded vesicles, we are creating a molecular barcoding and reporting system. This system helps us sort and enrich EVs with the desired drugs and confirm that EVs have delivered their cargo to the correct destinations. These therapeutic EVs hold great promise for improving the effectiveness and safety of delivering genetic medicine to treat genetic hearing loss, and we hope to pave the way for their use in clinical settings.
Watch a Q&A with Xiaoshu
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.
