UW researchers using gene editing to develop drugs for blindness

gene editing graphicUW-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.

“The genome editing piece of it is a game changer,” said Krishanu Saha, a professor of biomedical engineering at UW-Madison’s Wisconsin Institute for Discovery. “The opportunity to execute it in a safe and meaningful way for patients, specifically Wisconsin patients currently diagnosed with one of these diseases, would be a nice fulfillment of why we do the work and why it’s publicly funded.”

Wisconsin State Journal article