Vania Broccoli

Adeno‐associated virus ‐mediated restoration of Na V 1.1 in targeted brain regions ameliorates epilepsy in symptomatic Dravet syndrome mice

Abstract Objective Dravet syndrome (DS) is a developmental and epileptic encephalopathy primarily caused by haploinsufficiency of the SCN1A gene, which encodes the alpha subunit of Na V 1.1 voltage‐gated sodium channel. The disease manifests with febrile and spontaneous seizures,...

Targeted AAV gene therapy for neuroblastoma via direct capsid-antibody coupling

Abstract Adeno-associated viral (AAV) vectors are widely used in gene therapy for their versatility and safety, but their broad tropism limits cell-specific applications such as targeting primary or metastatic tumor cells. To address this, we developed AAV-STITCH, a strategy usin...