UW researchers using gene editing to develop drugs for blindness

UW-Madison researchers are part of a five-year, $29 million National Institutes of Health grant using gene editing to develop drugs for two rare diseases that cause blindness.

The project aims to use CRISPR technology, which stands for “clustered regularly interspaced short palindromic repeats,” to develop therapies for Best Disease, which affects older people’s central vision, and Leber Congenital Amaurosis, which affects children’s entire vision. The hereditary diseases are currently untreatable.

CRISPR, a “molecular scissors” that cuts DNA at certain locations and deletes or replaces sequences, was discovered in 2012. The method has resulted in few useful drug therapies because it’s hard to modify enough cells and the viral delivery system currently used can have side effects, such as immune reactions, a UW-Madison statement said. To overcome these limitations, the new project aims to use nanotechnology to develop efficient drug delivery systems.

Read the full article at: https://madison.com/news/local/business/health-care/uw-researchers-using-gene-editing-to-develop-drugs-for-blindness/article_fe3eb982-04a1-11ee-9e06-13c26f813cae.html