Giuseppe Ronzitti

In 2013, after a PhD in Biochemistry and a first post-doc in neuroscience, I joined the laboratory of Dr. Mingozzi at Genethon, to work on the development of gene therapy approaches for rare genetic diseases. Since then, the primary objective of my research was to overcome the main hurdles of AAV gene therapy i.e. immunogenicity, off-targeting and cargo capacity. I contributed to the development of gene therapy approaches for Crigler-Najjar syndrome and Pompe disease, that are now in the clinic and other projects are in advanced preclinical development. I also participated in important articles describing methods to reduce the impact of immunogenicity on gene transfer with AAV vectors.

I am currently an INSERM researcher affiliated to U951 ’’INTEGRARE’’ led by Dr. Anne Galy and head of the Immunology and Liver gene transfer team at Genethon.