Gene Therapy
The goal of The Lowy Medical Research Institute is to advance new therapies to treat Macular Telangiectasia type 2. There is a significant research effort underway to identify the genetic cause of MacTel, as well as to identify the factors that can promote photoreceptor survival in MacTel. LMRI supports a gene therapy research program so that we are prepared to translate the discoveries made in the laboratory to the clinic.
In gene therapy, viruses are used in a controlled fashion to deliver new genes into cells. When a virus infects a cell, it injects DNA into the cell. Scientists can engineer viruses that will infect only specific types of cells, and that will inject a specific piece of DNA that will help the patient. In this way, scientists are theoretically able to “fix” cells, causing the cells to express a new gene that improves their function. The viruses used for gene therapy don’t cause disease.
The gene therapy research that LMRI supports focuses on developing adeno-associated vectors (AAV) that can deliver genes to specific cells in the eye. The use of AAV for gene therapy is a technique that has been demonstrated to be relatively safe and effective. These vectors have been used to treat inherited eye diseases in more than 250 patients. Dr. John Flannery at University of California, Berkeley heads the LMRI gene therapy research program.