Giuseppe Ronzitti

Giuseppe Ronzitti

Company: Genethon

Job title: INSERM Researcher,
Head of Immunology and Liver Gene Transfer Laboratory


Development of a Liver-Based AAV Gene Therapy for Pompe Disease from the Bench to the Bedside 3:00 pm

Optimization of the liver secretion enhances the targeting at whole body level Comparison with enzyme replacement therapy define AAV gene therapy efficacy at very low doses Preclinical optimization enable clinical translation of the approachRead more

day: Day Two

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