Pasquale Piccolo

Pasquale Piccolo is an Assistant Investigator at the Telethon Institute of Genetics and Medicine (TIGEM) in Naples, Italy, specializing in liver-directed gene therapy for genetic disorders with liver fibrosis. He earned his Ph.D. in Human Genetics from the University of Naples 'Federico II' and conducted postdoctoral research at Baylor College of Medicine under Philip Ng.

• Ph.D. in Human Genetics (University of Naples 'Federico II')
• Bachelor in Medical Biotechnology (University of Naples 'Federico II')

His research focuses on overcoming fibrosis barriers in AAV-mediated gene therapy, developing dual AAV systems and targeted integration for Wilson Disease (ATP7B mutations). He investigates how fibrosis affects gene transfer efficiency and aims to engineer synthetic AAV vectors for improved hepatocyte transduction.

Recent publications highlight his work on nuclease-free genome editing, ATP7B trans-splicing, antifibrotic microRNAs (miR-34b/c), and fibrosis-induced barriers to AAV vectors. His studies bridge vector engineering, molecular biology, and rare disease treatment.

• α1-Antitrypsin Laurell's Training Award
• Gordon L. Snider Scholar Award

Piccolo mentors PhD students Maria Battipaglia, Simone Pisano, and Teresa Imperatore, while collaborating with postdoctoral fellows and researchers in his lab. His work is supported by grants from the EC-AVolution consortium, Alpha1-Foundation, and PFIC Network.