Evaluation of cAbl allosteric inhibitors in Gaucher-Parkinson

National Center For Advancing Translational Sciences

Investigators

Abstract

The main goal of this collaboration is to understand the role in cAbl kinase in the neurodegeneration associated with GBA1 mutants in Parkinson disease and evaluate cAbl allosteric modulators as potential treatment for this disease using mouse models. During this period, the collaborative team developed a research plan to interrogate the role of cAbl in neurogenesis, including animal models of Parkinsons. Initial efficacy studies in murine models of A53T SNCA and GBA1-associated Parkinson disease were completed, and promising activity was observed.