A New Approach to a Rare, Incurable Form of Blindness

A collaboration among four McPherson ERI member labs has resulted in a proof-of-concept method to correct an inherited form of macular degeneration that causes blindness, and that is currently untreatable.  The research, which focused on Best disease and was led by Divya Sinha, an Assistant Scientist in David Gamm’s lab, and Ben Steyer, an MD-PhD student in Kris Saha’s lab, demonstrated that a two-tiered gene therapy strategy may be able to treat all Best disease mutations in a highly effective manner.  An in-depth account of their work is in the McPherson ERI Summer 2020 InSights.


Read the full article at: https://www.med.wisc.edu/news-and-events/2020/july/researchers-devise-new-treatment-for-best-disease/