Research in the Grousbeck Gene Therapy Center and Vandenberghe Lab is focused on developing and translating genetic therapies to the clinic with an emphasis on blindness and other neurosensory disorders. We aim at defining, characterizing and overcoming hurdles on the path of gene therapies to clinical applications through
- The study of the biology of, and host response to genetic therapy.
- The development of enabling gene transfer technologies.
- The translation of specific therapeutic programs.
Our laboratory is located at the Schepens Eye Research Institute and Massachusetts Eye and Ear Infirmary, both affiliates of Harvard Medical School. We are a proud member of the Ocular Genomics Institute, whose mission is to define and treat inherited retinal forms of blindness.