- Prof. Stocco Gabriele
CV
ABSTRACT
Pediatric pharmacogenomics aims to optimize therapy by accounting for genetic and developmental factors that influence drug response. However, mechanisms underlying variability in efficacy and toxicity remain incompletely understood. Extracellular vesicles (EVs), secreted by all cell types, have emerged as key mediators of intercellular communication and carry cargo that mirrors the genetic and physiological state of the parent cell. Our group has investigated EVs as translational tools to better understand drug response in children.
We recently demonstrated that neuronal-derived EV microRNA profiles can predict susceptibility to ketamine-induced adverse events, providing a potential strategy for safer pediatric sedation. In parallel, we studied EVs from children with inflammatory bowel disease treated with thiopurines and relevant in vitro models, identifying how PACSIN2 genetic variants alter EV content, thereby modulating thiopurine sensitivity. These findings highlight EVs both as mechanistic links between genetic background and drug response, and as minimally invasive biomarkers for precision therapy.
By integrating pharmacogenomics with pharmacological research, this approach offers novel opportunities to improve therapeutic drug monitoring and advance individualized pharmacotherapy in pediatrics.

