Role of UBE3A in the Developing Brain

Your ASF donations hard at work at UNC and Erasmus Medical Center in the Netherlands. This pivotal study demonstrated that UBE3A is most critical in the developing brain and plays a less vital role later on in life.

This is important in designing clinical trials and knowing when is the best time to treat. But what does this mean for our loved ones? “This means that while developing a therapy that permanently reinstates UBE3A might be optimal, even a treatment that gives back UBE3A during critical periods of brain development could be truly transformative!” – Ben Philpot, PhD University of North Carolina at Chapel Hill