The basis of gene therapy research within our department is the use of genetic material, whether in the form of DNA or RNA, to accomplish therapeutic changes in the physiology of our patients. Our knowledge of the human genome provides us with the blueprints to change human biology and, importantly, provide a means to treat or prevent disease.
The Weill Cornell Department of Genetic Medicine has an enduring history of accomplishment in the field of gene therapy. Before coming to Weill Cornell Medical College in 1993, our department chair, Ronald G. Crystal, was the first physician to administer a genetically modified, replication-deficient virus to a human patient in an approved clinical trial. At Weill Cornell, investigators have continued this innovative legacy with gene therapy breakthroughs including:
- the first gene therapy experiment to grow blood vessels in human hearts
- the first gene therapy clinical trial for patients with neurodegenerative Batten Disease
- recently, the first gene therapy model for the treatment of peanut allergy
Our investigators are now using gene transfer strategies to develop therapies for clinically significant disorders including:
- Alzheimer’s Disease
- alpha-1 antitrypsin deficiency
- glioblastoma multiforme (GBM)
- cocaine addiction
