Researchers in Lab  UNC scientists in the lab, dedicated to finding a cure for Angelman syndrome, work tirelessly on ground-breaking research.

DNA Strand  Angelman syndrome is caused by mutations in the maternally inherited UBE3A gene, crucial for brain development.

Child with Angelman Syndrome  Children with Angelman syndrome face challenges such as poor muscle control, limited speech, and intellectual disabilities.

Ben Philpot in Lab  Ben Philpot, a leading expert, and his team have identified a small molecule that could activate the dormant gene.

Lab Equipment and Test Tube Over 2,800 small molecules were screened to find one that could effectively treat Angelman syndrome.

Mouse Model  The molecule (S)-PHA533533 showed excellent uptake in the developing brains of mouse models.

Hanna Vihma with Research Team  Hanna Vihma, a postdoctoral research fellow, plays a key role in the promising study published in Nature Communications.

Molecular Structure  The identified small molecule could noninvasively turn on the dormant paternally inherited UBE3A gene brain-wide.

Hopeful Family  This breakthrough offers new hope for families affected by Angelman syndrome, bringing them closer to an effective treatment.