Aleksandr Taranov was a neuroscience graduate student in Agnes (Yu) Luo’s lab at the University of Cincinnati when he helped test a new gene therapy that could one day change how doctors treat hydrocephalus — a condition where too much fluid builds up in the brain. Right now, treatment means brain surgery to insert a shunt, a small tube that drains the fluid. But these shunts fail in nearly half of patients within two years, often requiring emergency follow-up surgeries. The new therapy, tested in animal models, uses a harmless virus to deliver genetic instructions that reduce the brain’s production of this fluid at the source.
Hydrocephalus affects about 1 in every 1,000 babies born in the U.S., and can also strike adults after brain injuries or infections. Left untreated, the pressure from excess cerebrospinal fluid can cause seizures, developmental delays, and even death. For decades, treatment has relied on shunts or partial cauterization of the choroid plexus — the brain tissue that produces the fluid. But both options are risky and often temporary. The new approach offers hope for a more lasting, less invasive fix.
The therapy uses an adeno-associated virus, or AAV, a tool commonly used in gene therapy to deliver genetic material into cells. In this case, it targets the cells in the choroid plexus, reducing their ability to produce cerebrospinal fluid. In tests on animal models, the treatment not only lowered fluid production but also prevented brain swelling. When researchers at UC Davis induced hydrocephalus in adult animals, the therapy successfully stopped excess fluid from spreading into brain tissue. MRI scans later confirmed a clear reduction in swelling.
The collaboration between Cincinnati and UC Davis strengthened the study’s results. Cameron Sadegh and Olga Chechneva, neurosurgery researchers at UC Davis, led the animal testing, while Luo’s team developed the therapy. A blinded analysis of the MRI data confirmed the findings, adding scientific rigor. This builds on Luo’s earlier 2024 study, where her team first showed that selectively ablating choroid plexus tissue in adults could reduce fluid buildup — a breakthrough that opened the door to gene-based treatments.
While still years from human trials, the therapy could eventually be delivered through an existing shunt, making it minimally invasive. "This would allow us to have a minimally invasive and feasible treatment for hydrocephalus," said Taranov. For families facing repeated surgeries, that possibility brings real hope. The work also deepens scientists’ understanding of how hydrocephalus damages the brain — knowledge that could lead to even more treatments. As research moves forward, one thing is clear: a new chapter in brain health may be starting in a lab in Cincinnati.