About the Lab
Broadening Therapeutic Gene Transfer Through Mechanistic Studies, Technology Development, and Translation to the Clinic
Gene therapy is a conceptually naïve approach that has proven difficult in execution. It is the bringing together of a molecular intervention to a disease target by the delivery of a gene. Over 40 years in the making, clinical studies over the past years have illustrated the transformative potential of gene therapy in inherited blindness, hemophilia, and motor neuron disease.
Research in the Vandenberghe laboratory is focused on broadening applications of gene therapy generally. Research is centered on three overlapping areas of focus: Mechanism, Technology, and Translation.