Center For Technology Commercialization

Researchers have identified a GCase protein with a specific mutation that increases the protein’s binding in the lysosome. Inside the lysosome there is a receptor called “Lysosomal Integral Membrane Protein 2” (LIMP-2). This receptor is the “docking station” for the GCase protein. Lysosomal storage disorders and neurological disorders have problems with the Gcase protein binding to the LIMP-2 receptor. Researchers have found that this mutated GCase protein aids in increased GCase secretion from cells, and therefore increased binding to the LIMP-2 receptor in the lysosome. In addition, increased secretion could mean the ability to supply large amounts of secreted enzyme for therapeutic effects both in local and distant parts of the body. Such an approach would be particularly helpful in the CNS variants of Gaucher or Parkinson’s disease, which are caused by GCase defects within the body.