Abstract
Neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, and the polyglutamine diseases, have been defined as a group of intractable disorders, which are characterized by the progressive degeneration of neurons in various regions of the brain, resulting in neurological and psychiatric symptoms. Molecular genetics and biological studies have revealed that most neurodegenerative diseases are caused by protein misfolding and aggregation, and hence they are considered to belong to the so-called protein misfolding diseases. Moreover, recent emerging evidence has suggested that the misfolded protein aggregates formed in these diseases have similar intrinsic characteristics, i.e., they are propagated by prion-like infectious mechanisms. Therefore, various therapeutic strategies targeting protein misfolding and aggregation are being extensively explored. Here we introduce emerging disease-modifying therapeutic approaches against neurodegenerative diseases, particularly those targeting the misfolding and aggregation of toxic proteins. The development of anti-misfolding and anti-aggregation agents that are commonly effective against a wide range of neurodegenerative diseases is eagerly anticipated in the near future.
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Acknowledgments
We thank Dr. H. Akiko Popiel for critical reading of the manuscript, and lab members for their helpful discussions. This work was supported in part by Grants-in-Aid for Scientific Research on Innovative Areas (Synapse and Neurocircuit Pathology to Y.N.) and Strategic Research Program for Brain Sciences (Integrated research on neuropsychiatric disorders to Y.N.) from the Ministry of Education, Culture, Sports, Science, and Technology, Japan; by Grants-in-Aid for Scientific Research (B) and Challenging Exploratory Research to Y.N., and for Young Scientists (B) to E.N.M. from the Japan Society for the Promotion of Science, Japan; by Health Labour Sciences Research Grants for Research on Development of New Drugs and Research on Measures for Intractable Diseases to Y.N. from the Ministry of Health, Labour and Welfare, Japan; and by a grant from Core Research for Evolutional Science and Technology (CREST) of the Japan Science and Technology Agency to Y.N.
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Nagai, Y., Minakawa, E.N. (2015). Drug Development for Neurodegenerative Diseases. In: Wada, K. (eds) Neurodegenerative Disorders as Systemic Diseases. Springer, Tokyo. https://doi.org/10.1007/978-4-431-54541-5_9
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