Blocking an Enzyme Restored Brain Growth in Angelman Syndrome Mice

In mice modeling Angelman syndrome, elevated asparagine synthetase triggered a neuronal stress response that stunted brain growth. Blocking the enzyme's production restored normal brain growth and healthy behaviors. The study was published in Nature.

• Angelman syndrome affects about 1 in 20,000 births
• Without UBE3A, 23 proteins accumulated; asparagine synthetase stood out
• Blocking the enzyme restored brain growth and behavior in mice
• Drugs targeting the stress response remain a distant goal