Gene therapy offers new hope for hydrocephalus patients

Hydrocephalus happens when excess cerebrospinal fluid builds up in the brain, increasing pressure and damaging nerves. The usual fix is shunt surgery to drain fluid, but shunts often fail or require replacement. Endoscopic cauterization of the choroid plexus can help, yet it only partially reduces fluid and has safety limits. Researchers at the University of Cincinnati are testing a less invasive method that targets the cells that make fluid.

AAV gene therapy targets fluid‑producing cells

They used an adeno‑associated virus (AAV), a viral vector that delivers therapeutic genetic material into cells. The vector homes to choroid plexus cells and reduces their production of cerebrospinal fluid. In mice, the therapy lowered ventricular volume and prevented fluid from leaking into brain tissue. Because the virus can be injected through a shunt, the treatment could avoid open surgery.

UC Davis neurosurgeons made hydrocephalus in adult animals, then applied the gene therapy. MRI scans showed less brain swelling and a moderate drop in new neuron formation. The authors plan to test the therapy in larger animals and human tissues before human trials.