Vania Broccoli

This study develops Syn-specific CAR iTregs that reduce neuroinflammation and proteinopathy in PD mice by selectively targeting aggregated α-Synuclein.

This study introduces TES, an epigenetic silencer targeting YAP/TAZ-TEAD axis in GBM, which suppresses tumor growth and invasiveness while being safe for normal cells.

This paper develops a fully human stem cell-derived BBB model to study disease mechanisms and evaluate neurotropic AAV capsids.

This paper develops AAV-STITCH, a method to target neuroblastoma cells using anti-GD2 ScFv coupled to AAV capsids, showing selective transduction and tumor suppression in mouse models.

This study investigates how a WFS1 splice site mutation affects β cell apoptosis in Wolfram syndrome by analyzing alternative splicing and functional consequences in patient-derived iPSCs.

This study genetically modifies human pluripotent stem cells to generate lineage-restricted undifferentiated stem cells that efficiently produce mesencephalic dopaminergic neurons for potential clinical applications.

This study identifies MCT1-dependent metabolic failure and neuroinflammation as key factors in optic nerve degeneration in Wolfram syndrome mice, suggesting therapeutic targets for early intervention.

The paper shows that SET levels influence enhancer usage during cell fate acquisition, leading to altered gene regulation and contributing to disease.

This study identifies SULT4A1 as a regulator of synaptic development and function by modulating dendritic morphology and NMDAR synaptic activity through its interaction with Pin1.

AAV-PHP.eB-mediated delivery of an instability-prone Mecp2 transgene improves behavioral and molecular defects in Mecp2 mutant mice without severe toxicity.

This study uses CRISPRa to increase Kv1.1 expression in mouse hippocampal neurons, reducing seizures and cognitive deficits in a temporal lobe epilepsy model.