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

[NFS-02-5] iPS cell-based therapy for Parkinson's disease

Jun Takahashi (Center for iPS Cell Research and Application, Kyoto University, 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.

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.