Immunological Responses to AAV Gene Therapy in Neurological Compartments: Mechanisms and Management

November 30, 2022 | 1 - 3 p.m. CT

The full potential of AAV gene therapy is limited by immune responses that impact both safety and efficacy. An understanding of the mechanisms that mediate humoral and cellular immune responses may facilitate management of their effects in the clinic and improve outcome after administration of AAV gene therapy. This is particularly important for neurological compartments given the lack of neuronal regeneration and potential permanence of negative impact. Toxicity in the dorsal root ganglia (DRG) has been highlighted as a significant concern; insights into potential immunological mechanisms driving this toxicity are critical for effective amelioration. In clinical trials, the management of immunological responses has led to improved tolerability as well as efficacy in some cases, but more remains to be done to both characterize and understand immune responses in the clinic and effectively address them.

This session will provide an update on the humoral and cellular immune responses, including the impact of vector design, purity, and dose; DRG toxicity and potential immunological mechanisms; and management of immunological responses with a focus on the nervous system and implications for re-dosing.

This event is free for ASGCT Members and $50 for non-members.

Registrants & ASGCT Members: Watch On Demand


Moderators: Dinah Sah, PhD and Eva Andres-Mateos, PhD (Atsena Therapeutics)

Innate and Adaptive Immune Responses to AAV Gene Therapy

Allison Keeler-Klunk, PhD
University of Massachusetts

DRG Toxicity and Potential Immunological Mechanisms

Steven Gray, PhD
University of Texas Southwestern

Management of AAV Immune Response and Implications for Redosing

Shari Gordon, PhD

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