[NFS-02] The emergence of a new era of gene therapy and regenerative medicine in Neurology

[NFS-02-3] Gene therapy for Parkinson's disease

Shin-ichi Muramatsu^1,2,3 (1.Division of Neurology, Department of Medicine, Jichi Medical University, Japan, 2.Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo, Japan, 3.Department of Neurology, Osaka University Graduate School of Medicine, Japan)

In 2017, an antisense oligonucleotide, nusinersen, was approved as the first treatment for spinal muscular atrophy (SMA). Nusinersen avoids the blood-brain barrier by being injected directly into the cerebrospinal spinal fluid space, and it also eclipsed the existing paradigm of neurological therapeutics. In addition, positive results of a clinical trial of SMA gene therapy using a neurotropic adeno-associated virus (AAV) type 9 were also announced. In 2018, furthermore, transplantation treatment using iPS cells / ES cells against Parkinson's disease was about to begin as an actual clinical trial.
At this symposium, world-leading scientists will talk about gene therapy / regenerative medicine with new modalities designed to overcome intractable neurological diseases.

He is currently a Professor of Neurology at Jichi Medical University (JMU), and a Project Professor of Center for Gene & Cell Therapy at The Institute of Medical Science, The University of Tokyo. He is also a Guest Professor of Department of Neurology, Osaka University. He received his M.D. and Ph.D. from JMU. He completed his neurology residency at Gunma University and JMU. He was a visiting associate at National Institutes of Health in USA from 1995 to 1997. He received the Top Abstract Award from the American Society for Gene and Cell Therapy (2009), He also received the Award for Excellent Research (2001) and the TAKARA Bio Award (2011) from the Japan Society for Gene and Cell Therapy. He is a founding member of Gene Therapy Research Institution Co., Ltd.