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

[NFS-02-1] Gene therapy with therapeutic oligonucleotide

Tetsuya Nagata (Department of Neurology and Neurological Science, 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.

2018-Current
Research Associate Professor, Department of Neurology, Department of Neurology and Neurological Science, Tokyo Medical and Dental University
2014-2018
Research Senior Assistant Professor, Department of Neurology, Department of Neurology and Neurological Science, Tokyo Medical and Dental University
2009-2014
Section chief, Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry
2008
Assistant Professor, Department of Neurology, Okayama University School of Medicine
2005-2008
Postdoctoral fellow, Department of Neurology/Center for Motor Neuron Biology and Disease, Columbia University
2003-2005
Assistant Professor, Department of Neurology, Okayama University School of Medicine
1999
Graduated from Tohoku University Graduate School
1994
Graduated from Tohoku University School of Medicine