‘IPS-derived Neural Precursor Cells to Study Mechanism and Identify Modulators of Parkinson?s Disease’

Richard Wade-Martins and Sally Cowley, Oxford University; Jesús Madero-Pérez and Pilar Rivero-Ríos, Consejo Superior de Investigaciones Científicas (CSIC)

Parkinson’s disease (PD) is a debilitating neurodegenerative disorder without cure. Recent genetic findings highlight the importance of leucine-rich repeat kinase-2 (LRRK2) in both familial and sporadic forms of PD. pathogenic LRRK2 function is amenable to drug targeting strategies, but this requires defined cellular readouts.

The present project will investigate the effects of pathogenic LRRK2 on centrosomal, Golgi and nuclear shape alterations in the context of neural degeneration, using iPS-derived neural precursor cells from mutant LRRK2 PD patients, sporadic PD patients and healthy controls.

This cellular disease model will further be employed to study disease mechanism(s) and analyze the effect of various compounds for their ability to modulate degeneration of the human patient-derived neurons.