Press Releases & Research Commentary

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New Platform Combines Precision Gene Targeting with Brain-Wide Delivery

Wednesday, July 8, 2026

A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain. The approach addresses two major challenges in neurological medicine—reaching therapeutic targets behind the blood-brain barrier and limiting unwanted effects elsewhere in the body—and could pave the way for new treatments for diseases including multiple sclerosis, Huntington's disease, and rare childhood white matter disorders.

The platform pairs specially engineered adeno-associated viruses (AAVs) with a delivery strategy that harnesses the brain's natural fluid transport pathways. Together, these innovations enabled researchers to deliver therapeutic genes broadly throughout the brain, preferentially targeting human glial cells while minimizing exposure to other cell types and organs.

“Gene delivery to the brain has always faced two major obstacles,” said Steve Goldman, MD, PhD, co-director of the University of Rochester Medicine Center for Translational Neuromedicine and lead author of the study, which appears in Nature Biotechnology. “You need a way to get therapies into the brain selectively and efficiently, and you need vectors that can deliver those therapies to the right cells once they get there. This work addresses both challenges simultaneously.”

Researchers Harness the Body’s Internal Clock to Improve Stroke Recovery

Monday, June 15, 2026

A new study from scientists at University of Rochester Medicine suggests that reinforcing the body's natural daily rhythms to improve sleep could help the brain recover after a stroke, pointing to a potential new strategy to improve brain waste clearance and outcomes long after the initial injury.

The research, which was published in the Journal of Clinical Investigation, found that interventions designed to reinforce the body's natural circadian rhythms improved recovery in mouse models of stroke. The benefits were accompanied by improvements in the glymphatic system—the brain's waste-clearing network—and reductions in inflammatory molecules that can linger in the brain after a stroke.

The findings build upon more than a decade of pioneering research led by URochester Medicine neuroscientist Maiken Nedergaard, MD, DMSc, whose laboratory discovered the glymphatic system in 2012. The system circulates cerebrospinal fluid through the brain, helping clear waste products and other debris. Subsequent research revealed that glymphatic activity is most robust during sleep and plays an important role in maintaining brain health.

Building on that discovery, neuroscientist Lauren Hablitz, PhD, helped demonstrate that glymphatic activity is governed not only by sleep but also by circadian rhythms—the body's internal 24-hour clock. In a landmark 2020 study, Hablitz, Nedergaard, and colleagues showed that glymphatic function follows daily rhythms independent of sleep itself, helping establish a direct connection between the brain's waste-clearing system and circadian biology.

Why is sleep so important? Your brain depends on it

Monday, January 26, 2026