Two scientists review computer screen

Our Research

The Gene Therapy Institute brings together a vibrant and dynamic community that fosters a collaborative approach to address the unique clinical challenges of gene therapy research, vector development & production, device & delivery engineering, operational efficiency, physician training and innovative business models to ensure maximal patient benefit.

Research Areas

Neurons

Nervous System Diseases

nanocarrier uptake by cells

Non-Viral Therapies

Dr. Sy Moroi standing in a lab

Ophthalmology

The Gene Therapy Institute is truly trans-institutional and designed to accelerate basic science discoveries into clinical cures for untreatable, or incompletely treated, disorders.

Russ Lonser, MD
Director, Gene Therapy Institute

Support the Institute

A gift to the Gene Therapy Institute can take us closer to new discoveries and life-changing treatments.