UW researchers use gene editing to create drug therapies for blindness
As part of a five-year, $29 million grant from the National Institutes of Health (NIH), UW–Madison researchers are using gene editing to develop drug therapies for two rare diseases that cause blindness, the Wisconsin State Journal reports.
The project uses Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology to address Best Disease and Leber Congenital Amaurosis, which affect older people and children, respectively. Both diseases are hereditary and currently untreatable.
The new project uses nanotechnology to develop efficient drug delivery systems. It is part of the NIH’s Somatic Cell Genome Editing Consortium.