06 Jun UW researchers will trial gene editing therapy to treat blindness

By Laura Red Eagle, UW–Madison

With new support from the National Institutes of Health, a team of researchers at the Wisconsin Institute of Discovery will lead drug therapeutics testing for two diseases known to cause blindness.

Over the next five years, the collaborative project will use the $29 million NIH grant to merge new drug delivery systems with advanced genome CRISPR technology, innovating new treatments for Best Disease (BD) and Leber Congenital Amaurosis (LCA), both of which are currently untreatable hereditary diseases.

“Genetic mutations can cause some of the most rare and devastating disorders of the nervous system,” said Walter Koroshetz, co-chair of the Somatic Cell Genome Editing Program and director of the National Institute of Neurological Disorders and Stroke. “Thanks to large-scale efforts like the SCGP, we are starting to bring tools into the clinic to edit out these gene mutations. While there are still challenges to overcome, the level of hope for effective treatments is high.”

The researchers decided to focus on the eye as their starting point because it is self-contained and isolated from other organs as well as for its accessibility, ease of monitoring and reduced likelihood of adverse immune reactions. Read more …