Giuseppe Ronzitti, PhD (Short CV)
Genethon, INSERM U951 INTEGRARE,
University of Evry,
University Paris-Saclay, FranceAbstract
Liver gene transfer with AAV vectors has become a clinical reality. However, limitations remain that preclude the use of AAV vectors in larger patient populations. Through my presentation I will discuss the strategy of my team, oriented to the overcoming of current AAV gene transfer limitations. In particular I will discuss how we are tackling two important AAV limitations i.e. the humoral immunity and the toxicities associated to liver overloading in neuromuscular diseases. In particular I will discuss the recently reported use of IdeS endopeptidase to degrade anti-AAV immunoglobulins as well as show unpublished data on the development of new AAV capsids with a methodology developed in our laboratory.