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

[NFS-02-4] Gene therapy against SCA1 based on the molecular pathomechanism

Hitoshi Okazawa^1,2 (1.Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, Japan, 2.Center for Brain Integration Research, Tokyo Medical and Dental 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.

Dr. Hitoshi Okazawa graduated from The University of Tokyo and received license of medical doctor in 1984. After clinical training as a resident in The University of Tokyo Hospital, he became a staff member of Department of Neurology The University of Tokyo in 1986. He started research of molecular biology at Department of Biochemistry The University of Tokyo in 1988, and received Ph.D. by discovery of Oct-3/Oct-4 in 1991, which is later recognized as the gatekeeper of ES cell differentiation and as the most important factor for iPS cell generation. He then moved to Max-Planck Institute of Psychiatry in Munich Germany as a staff scientist in Department of Neurobiochemistry, where he discovered a novel receptor for brain-derived neurotrophic factor (Development 1994). He moved to Tokyo Metropolitan Institute for Neuroscience as Department Chair in 2001. He has been Professor and Chair of Neuropathology TMDU from 2003, Director of Center for Brain Integration Research TMDU from 2012.