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Global Science Summit: Pillar 2 Sneak Peek

What is Pillar 2?

Pillar 2 encompasses all work being done to "turn on dad's gene" by activating the silent paternal copy of the UBE3A gene in neurons. To explain briefly, everyone has two copies of the UBE3A gene—one from their mom and one from their dad. In the brain, the dad's copy is usually turned off because of an antisense transcript that binds to it, stopping the process needed to make UBE3A protein. In Angelman syndrome, where the brain lacks functional UBE3A, a treatment that can activate dad's UBE3A offers a promising therapeutic approach.

Pillar 2 Speakers

The Tale of Two Therapies for Angelman Syndrome

Watch the 2023 presentation from Dr. Segal:

Non-Viral Gene Editing for Angelman Syndrome: Progress and Updates

Watch the 2023 presentation from Drs. Jiang and Zhou:

ETX201: A Vectorized miRNA-based Approach for the Potential Treatment of Angelman Syndrome

Watch the 2023 presentation on ETX-201:

Ionis’ Program Update of ION582 for Individuals Living with Angelman Syndrome

Watch the 2023 presentation from Dr. Crean:

A GTX-102 Clinical Development Update: New Results From the Phase 1/2 Open-label Study and an Overview of the Pivotal Phase 3 Study

Watch the 2023 presentation on GTX-102:

Angelman Syndrome News