Webinar: Dual-AAV Gene Therapy Corrects Dravet Syndrome in Mice

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Abstract

Join Dr. Franck Kalume for a webinar exploring a major breakthrough in gene therapy for Dravet syndrome (DS), a devastating childhood epilepsy disorder. In a recent collaborative study between Seattle Children’s Research Institute and the Allen Institute, researchers successfully developed an interneuron-specific dual-AAV gene replacement strategy that restored SCN1A function and rescued epileptic phenotypes in mouse models of DS.

This novel approach leverages split-intein technology and a dual-vector AAV system to overcome size constraints of the SCN1A gene, achieving functional expression of NaV1.1 channels specifically in GABAergic interneurons using the DLX2.0 enhancer. The result: complete prevention of premature death, significant seizure reduction, and long-term therapeutic benefits without off-target effects or toxicity.

Dr. Kalume will walk attendees through the design, execution, and implications of this cutting-edge study, including comparisons between cell-specific and pan-neuronal gene delivery. This research not only provides a promising disease-modifying strategy for DS but also sets a new precedent for precision-targeted AAV therapies in neurogenetic disorders.

Time

Aug 6, 2025, Wed, 2:00-3:00 pm EDT, 11:00am -12:00pm PDT

Speaker:

  • Kalume

    Name: Franck K. Kalume, Ph.D.

    Company: Seattle Children’s Research Institute; Department of Neurological Surgery, University of Washington

    Title: Associate Professor of Neurological Surgery, Adjunct Associate Professor of Global Health, Adjunct Associate Professor of Pharmacology

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