Immune Responses to Gene Therapies for Musculo-Skeletal and Cardiac Diseases

February 23, 2022 | 1:00-3:00 p.m. (CT)

This session highlights the ongoing research related to the administration of high-dose adeno-associated virus (AAV) in patients with an emphasis on safety and tolerability findings. Speakers will provide a better understanding of the responses of patients to systemic delivery of AAV and the array of related factors.

Registrants & ASGCT Members: Watch On Demand


Reactions to High-Dose Systemic AAV Delivery in DMD Patients

Juliette Hordeaux, DVM, PhD
University of Pennsylvania

Lee Sweeney, PhD
University of Florida

Louise Rodino-Klapac, PhD
Sarepta Therapeutics

Serge Braun, PharmD, PhD


Register for the ASGCT Policy Summit

September 23-24, 2024 | Washington, D.C.

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