Intracisternal AAV9-based Gene Therapy for Lysosomal Storage Diseases: The Clinical Trial Experience

Time: 8:35 am
day: Day Two


• Intracisternal route of administration in pediatric MPS I and II patients
• Safety and monitoring of intracisternal AAV9 gene therapy
• Biomarker and clinical outcomes assessment challenges in heterogenous phenotypes of MPS II
• Progress and update of the first in human AAV9 gene therapy studies for MPS I and II