The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $789,088 Project Grant (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to Childrens Hospital Medical Center in Cincinnati, Ohio to evaluate a novel, less invasive tool for ablating the choroid plexus to treat neonatal hydrocephalus. The project aims to rigorously assess the efficacy and safety of an adeno-associated virus (AAV) vector targeting the choroid plexus and delivering a suicide gene to ablate this primary cerebrospinal fluid production organ. The study will leverage human choroid plexus organoids, rodent models of neonatal hydrocephalus, and advanced neuroimaging to determine the pharmacokinetics, off-target effects, and therapeutic potential of this new treatment approach for pediatric hydrocephalus, which currently relies on surgical interventions with significant limitations.
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