One of the nation’s 10 most prominent research institutions, UW–Madison is unique for its collaborations, which help researchers become innovators and bring life-changing advancements to patients. Thanks to a significant boost from federal funding and the generosity of dedicated donors, researchers at the UW are on the cusp of revolutionizing treatments for two rare, currently incurable eye diseases: Best disease (BD) and Leber congenital amaurosis (LCA).
The National Institutes of Health (NIH) awarded a $29 million grant involving the McPherson Eye Research Institute (MERI) and the Wisconsin Institute for Discovery (WID) to launch the CRISPR Vision Program. This ambitious initiative, led by WID and MERI/Retina Research Foundation (RRF) Kathryn and Latimer Murfee Chair and biomedical engineering professor Krishanu Saha, merges cutting-edge CRISPR gene-editing technology with novel drug delivery systems to develop therapies for BD and LCA, hereditary diseases that cause early and progressive vision loss.
David Gamm, ophthalmology professor and director of MERI, emphasizes the promise of the project. “By targeting these diseases,” says Gamm, “we gain a broader understanding of how gene editing can treat many types of genetic retinal disorders.” Gamm’s lab focuses on developing laboratory-based human retinal models using stem cell technology, a critical step toward ensuring safety and effectiveness before advancing to patient trials.
One key innovation driving this effort is the development of safe, efficient drug delivery systems. Traditional methods using viral vectors, which are modified viruses, carry risks such as immune reactions and unintended genetic effects. To overcome these challenges, Shaoqin “Sarah” Gong, Vilas Distinguished Professor, Advancing Vision Science Chair Professor, and RRF Edwin and Dorothy Gamewell Professor, is pioneering minuscule, man-made fragments called synthetic nanoparticles. These biodegradable particles safely carry CRISPR tools to target cells, without the long-term risks associated with viral delivery.
MERI Daniel M. Albert Chair and associate professor Bikash Pattnaik leads a collaborative project within the CRISPR Vision Program that includes engineers and scientists from the UW, Harvard, and MIT to work on an innovative technique to deliver gene-editing components into cells.
“Typically, drug development can take more than 30 years,” Pattnaik says. “But with a multidisciplinary approach that brings together people with different expertise, we can cut this timeline significantly.” He says this work is a critical first step toward restoring the sight of affected young patients.
The project’s success hinges on collaboration and investment, and private donor support has been a crucial catalyst. Years of foundational research — often too early-stage to attract traditional funding — have been sustained through donor generosity. This vital investment helped MERI and WID build the expertise and infrastructure necessary to compete for major federal grants like the NIH’s. Endowed chairs and professorships contributed by MERI namesake Alice McPherson ’48, MD’51, through the Houston-based RRF, founded years after earning her undergraduate and medical school degrees at UW–Madison, are also essential to making this work possible.
“Rare diseases often face a funding gap because they aren’t commercially attractive to big pharmaceutical companies,” says Gamm. “But thanks to donors who believe in advancing cures for all patients, we’re able to move innovative ideas toward real-world treatments.”
Saha sees this moment as a major leap forward. “The genome editing piece is a game changer,” Saha says. “Thanks to public investment — and the support of donors — we’re closer than ever to bringing meaningful treatments to patients who have been waiting too long. Success in this program could ignite drug development with CRISPR technology for many other retinal diseases.”
While these groundbreaking therapies are still in development and will take time to perfect, the collaboration’s momentum is undeniable. This project not only represents a step forward in treating blindness but also serves as a model for how visionary donor support combined with public funding can drive scientific innovation and change lives.