Research
The Department of Genetic Medicine is a leader in gene therapy research, being the first to use a recombinant virus as a vehicle for in vivo gene therapy. Our faculty collaborates across various departments at Weill Cornell Medical College and Cornell University to refine, control, and target gene expression, supporting the translation of our research into practical treatments. Our research aims to develop treatments that tackle diseases at their genetic roots and can be divided into three main areas:
- Discovery Research: We test new theories about diseases and explore innovative ways to control and deliver genes to cells and tissues. Our current focus is on using viral vectors to deliver redesigned therapeutic proteins with the potential to be more effective than the native proteins they replace.
- Development Research: We enhance existing research by incorporating new technologies for better gene control and delivery. This includes using RNA interference to target gene expression to specific organs and optimizing viral vector targeting for disease treatment.
- Candidate Therapies: We develop and translate research into gene therapies and vaccines. Our strategies include using DNA-based viral vectors to treat central nervous system disorders, including Alzheimer's and vision loss. Additional research is aimed at enhancing orthopedic repair, protecting against allergies, treating metabolic disorders, and preventing damage due to oxidative stress.
The overall goal of our translational research program is to utilize our expertise in the fields of genetic therapies and personalized medicine to produce clinical solutions that address the molecular causes of human disease.
