Overview of Gene-Based Therapies for Glaucoma

In this presentation from AGS 2017, Dr. Paul S. Kaufman provides an overview on investigational gene therapy targets to treat glaucoma. He describes steps of the development process, which is currently underway in several preclinical investigations.

• Strategy: Reprogram target cells to make more or less of something.
• Method: Create a viral vector incorporating the gene of interest.
• Consequences: Observe the up- or down-regulation of the biochemical/physiological process of interest.
• Obstacles: Examine the duration of expression, possible viral toxicity, efficacy, immune response, and localization of cell transfection.

He describes how researchers in this field are not interested in the pathophysiology of pressure abnormalities, rather, they focus on the pathways of therapeutic targets and how drugs work on them.