Delivery to Skeletal Muscles: Breaking through Barriers for Genetic Medicine

October 19, 2022 | 1 - 3 p.m. CT


Neuromuscular disorders represent a diverse family of diseases that affect both infants and adults. Gene therapy approaches have been gaining strong momentum and have the potential to provide durable and effective results. In this session we will explore use of gene therapy approaches to treat Pompe disease and discuss challenges as well as novel uses of AAV to treat neuromuscular disorders. We will hear discussions regarding how these developments impact the standards and metrics necessary to support clinical translation. The future of gene therapy is ever evolving and this Insight Series session will provide an excellent overview of both the challenges and the versatility of AAV for treating neuromuscular diseases.

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Agenda

Moderators: Christina Pacak, PhD and Eduard Ayuso, DVM, PhD
University of Minnesota, DiNAQOR AG

Gene Therapy Approaches for Late Onset Pompe

Dwight Koeberl, MD, PhD
Duke University Medical Center

Non-Viral Approaches for Editing Muscle Cells

Jacques Tremblay, PhD
University Laval and CRCHUQ

AAV-delivered Suppressor tRNA

Dan Wang, PhD
UMass Chan Medical School

Registration

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2022

ASGCT Policy Summit

Watch On Demand Through Oct. 27

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