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

[NFS-02-5] iPS細胞を用いたパーキンソン病治療

高橋 淳 (京都大学 iPS細胞研究所)

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.

Jun Takahashi is a professor and deputy director of CiRA, Kyoto University. He graduated from the Kyoto University Faculty of Medicine in 1986 and thereafter started his career as a neurosurgeon at Kyoto University Hospital. After he earned his Ph.D. from the Kyoto University Graduate School of Medicine, he worked as a postdoctoral research fellow at the Salk Institute (Dr. Fred Gage), CA, U.S.A., where he started research work on neural stem cells. In 2012, he became a full professor at CiRA, pursuing stem cell therapies for Parkinson’s disease patients.