By actively working in the clinic and in the laboratory, my research focuses on new therapies for inborn errors of metabolism. I have been the principal investigator of phase 1/2 liver-directed gene therapy clinical trials. As independent investigator, I was funded by NIH and ERC, and I received awards from ASGCT and ESGCT. I am currently Professor of Medical Genetics at Federico II University of Naples and Associate Investigator at the TIGEM, Italy.