Pasquale Piccolo is Group Leader at the Telethon Institute of Genetics and Medicine (TIGEM) in Pozzuoli, Italy. He earned his Ph.D. in Human Genetics from the University of Naples “Federico II” and completed postdoctoral training at Baylor College of Medicine in Houston (TX), where he specialized in liver-directed gene therapy. Upon returning to Italy, he continued his work at TIGEM, focusing on genetic liver diseases and therapeutic development. As an independent investigator, his research centers on the use of adeno-associated viral (AAV) vectors for in vivo gene replacement and genome editing, particularly in the context of liver damage and fibrosis. His contributions have helped to define the challenges and opportunities of gene transfer in diseased tissues. His work has received international recognition, including the α1-Antitrypsin Laurell’s Training Award, the Gordon L. Snider Scholar Award, and the Daniel Alagille Award, and is supported by both European and US funding agencies.