Federico Mingozzi

Biography

Federico Mingozzi heads a research group at Genethon which is working on the development of liver-targeted gene therapies for inherited diseases and on the characterization of immunity and tolerance in gene transfer. He is also Associate Professor at the University Pierre and Marie Curie in Paris. He received his Biology degree and Ph.D. In Biochemistry and Molecular Biology from the University of Ferrara, Italy. He also obtained a Master in Business Administration from Drexel University, United States. He spent over ten years in the United States at the Children’s Hospital of Philadelphia and University of Pennsylvania where he was involved in the early development of gene therapies based on the adeno-associated virus (AAV) vector platform. In his research, he characterized the mechanisms of immune tolerance induction mediated by AAV vector liver gene transfer. More recently he focused on the clinical development of AAV vector-based gene therapies for inherited diseases and on the study of immune responses directed against AAV vectors in human trials. He contributed to several first-in-human AAV gene transfer clinical trials for indications including hemophilia B, Leber’s Congenital Amaurosis, and lipoprotein lipase deficiency. He contributed to about 80 publications, including original research articles, reviews and commentaries, and book chapters.

Research Interest

liver-targeted gene therapies