[NFS-02-4] Antisense oligonucleotide therapy in spinocerebellar ataxia type 3 (SCA3)/Machado-Joseph disease
Spinocerebellar ataxia (SCA) is neurodegenerative diseases affecting mainly cerebellum and brainstem, and new subtypes and pathologies of SCAs have been continuously discovered in the world. Especially, SCA31 and SCA36 (nicknamed Asidan) were first found in Japanese, and their clinical and pathological features are quite unique compared with other SCAs. Recent discovery of repeat associated non-ATG (RAN) translation in the pathogenesis of hereditary SCAs carrying repeat expansion mutations has tremendously extended our understanding in the pathogenesis of SCA and other neurodegenerative diseases. Furthermore, the development of new therapy for SCAs have been extensively investigated in Japan and the world. The aim of this symposium is to learn and discuss the current clinical, pathological and therapeutic findings of SCAs for Japanese and Asian neurologists and neuroscientists.
Henry L. Paulson, M.D., Ph.D., is the Lucile Groff Professor of Neurology for Alzheimer's Disease and Related Disorders in the Department of Neurology at the University of Michigan. Dr. Paulson joined the U-M faculty in 2007, and he currently directs the Michigan Alzheimer’s Disease Center (MADC) and co-directs the U-M Protein Folding Diseases Initiative.
Dr. Paulson received his medical degree and doctorate in Cell Biology from Yale University in 1990. He then completed a neurology residency and neurogenetics/movement disorders fellowships at the University of Pennsylvania. In 1997, he joined the Neurology faculty at the University of Iowa, where he remained until 2007.
Dr. Paulson's research and clinical interests concern the causes and treatment of age-related neurodegenerative diseases, with an emphasis on polyglutamine diseases, Alzheimer's disease and frontotemporal dementia. In 1997, his lab described abnormal protein aggregates in the polyglutamine diseases, which now are recognized as a pathological hallmark in this important class of inherited diseases. Using test tube, cell-based and animal models, he has contributed to advances in the understanding of various neurodegenerative diseases. His lab also has helped pioneer the use of gene silencing methods as potential therapy for the many neurological disorders caused by "toxic" mutant genes.
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