Pathogenesis of and therapy for Parkinson's disease

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  • MOCHIZUKI HIDEKI
    Department of Neurology, Research Institute for Disease of Old Ages, Juntendo University School of Medicine

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  • パーキンソン病の病因究明と治療へのチャレンジ

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Dopaminergic neuron loss in the substantia nigra (SN) is the central pathogenic event in the brains of patients with Parkinson's disease (PD), although the mechanism (s) of cell loss is not fully understood. Because many factors have been implicated in the pathogenesis of PD, such as aging, inflammation, chemicals and genetic factors, the pathogenetic mechanisms are presumably heterogeneous. Abnormal protein accumulation including a -synuclein plays important roles in many types of PD as shown by the studies of familiar PD. As a new therapeutic strategy for PD, gene therapy and regenerative medicine have emerged aiming at neuroprotection of nigral neurons. Autosomal-recessive juvenile Parkinsonism (ARJP) is caused by mutations in the PARK2 gene coding for parkin and constitutes the most common familial form of PD. The majority of ARJP-associated parkin mutations are thought to be loss of function-mutations. Therefore, gene therapy using parkin may be crucial in the treatment of ARJP.

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