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Global Science Summit: Pillar 1 Sneak Peek

As we gear up for the upcoming FAST Global Science Summit, we would like to take a moment to revisit the four Pillars that form the foundation of FAST’s Roadmap to a Cure 2.0. We will review each Pillar, the progress shared during last year’s Summit, and the exciting new developments to look forward to at this year’s event. Let’s start with Pillar 1.

What is Pillar 1?

We will start with Pillar 1, “Replacing Mom’s Gene”. This Pillar encompasses all work being done to address deficits due to a lack of a functional maternal UBE3A copy in the brain. Pillar 1 consists of therapeutic programs that focus on replacing the missing or non-functional UBE3A gene, or protein, in the brain of individuals living with Angelman syndrome.

Pillar 1 Speakers

There are 4 speakers presenting on Pillar 1 and we look forward to hearing exciting updates this year from each presenter.

Backstage Passes to the Brain for Therapeutic Delivery

Watch the 2023 presentation on CPPs:

Hematopoietic Stem Cell Gene Therapy (HSC-GT) for the Potential Treatment of Angelman Syndrome

Watch the 2023 presentation on HSC-GT:

CRISPR Activation: How we are Addressing the Genes Outside of UBE3A for Individuals with Deletions

Watch the 2023 presentation from Dr. Ahituv:

GTP-220: A Gene Replacement Therapy for Angelman Syndrome

Watch the 2023 presentation from Dr. Wilson:

Angelman Syndrome News