Denise Sabatino

Company: The University of Pennsylvania & The Children’s Hospital of Philadelphia

Job title: Associate Professor of Paediatrics

Bio:

 

Seminars:

Panel Discussion: Assessing the Risk of Insertional Mutagenesis by AAV Gene Therapies 5:45 pm

This panel will be a structured conversation designed to answer key questions such as: What is the rate of integration of AAV vectors? What is the structure of the integrated viral sequences? Is the risk dependent on dose? What is the role of species differences when looking at preclinical data, will this translate to humans?…Read more

day: Day One

Preclinical Studies in Large Animal Models Provide Insights Into AAV Integration & the Potential for Genotoxicity 5:15 pm

Hemophilia A dogs treated with AAV vectors delivering the factor VIII transgene were followed for as long as 10 years after vector administration providing a unique opportunity to study AAV integration AAV integration studies in the hemophilia A dogs showed patterns of integration and clonal expansions containing integrated vector were detected in the absence of…Read more

day: Day One