ARTICLE | Targets & Mechanisms

A RIPping target in Gaucher's disease

February 13, 2014 8:00 AM UTC

Enzyme-replacement therapies are highly effective for most patients with Gaucher's disease but do little for those with the neurological childhood forms of the disease. Now, an Israeli and U.K. team has unlocked the mechanism of nerve destruction in neuronopathic Gaucher's disease and identified receptor-interacting serine-threonine kinase 3 as a new target.1

The team is investigating possibilities for generating inhibitors of the protein kinase that cross the blood-brain barrier (BBB)...