Fetal gene therapy could treat neurodegenerative disease

A gene therapy virally delivered in utero or postnatally could help treat an acute form of Gaucher's disease, according to a Nature Medicine paper from University College London researchers and colleagues.

Mutations in glucocerebrosidase (GBA; GCase) causes Gaucher's disease, including an acute, lethal childhood form of the disease that carries symptoms consistent with neurodegeneration. While enzyme replacement therapy manages symptoms in adults with a milder form characterized by enlarged spleen or liver, no treatments exist for the acute form as the enzyme cannot cross the blood-brain barrier...