Title: Development of Gene Therapy for Pompe Disease: Recent History

Date: Tuesday, November 7, 2023

Speaker:
Dr. Dwight Koeberl

Webinar Overview:
Dr. Koeberl will discuss the rationale for gene therapy for Pompe Disease. He will provide a summary of research focused on clinical development of gene therapy with a focus on potential risks versus benefits, as well as expectations regarding the clinical use of gene therapy.

Speaker Bio:
Dr. Koeberl joined the Division of Medical Genetics in Pediatrics at Duke University in 1999. His group has developed new gene therapy and genome editing for glycogen storage diseases, including von Gierke Disease and Pompe Disease. In January 2019 the Division initiated a clinical trial of gene therapy for Pompe Disease.

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