Researchers to Develop Gene Editing Therapy for Blindness

Researchers from the Wisconsin Institute for Discovery will develop gene editing therapeutics for two diseases that can cause blindness. The collaborative project is funded by the US National Institutes of Health.

The eye was chosen as the initial point of the study because it is self-contained and separated from other organs. It is also accessible, easy to monitor, and less likely to experience negative immunological reactions. The scientists will combine advanced genome CRISPR technology with new drug delivery systems to produce new treatments for untreatable hereditary diseases—Leber Congenital Amaurosis (LCA) and Best Disease (BD).

LCA is a rare and severe group that affects children's entire vision. Meanwhile, BD is a slow-onset disease that affects older individuals' central vision. By focusing on these diseases, the researchers can acquire a more comprehensive understanding of the efficacy of their gene editing treatments.

For more information, read the news release of the University of Wisconsin-Madison.