60th Annual Meeting of the Japanese Society of Neurology

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Neuroscience Frontier Symposium

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

Sat. May 25, 2019 8:00 AM - 10:00 AM Room 3 (Osaka International Convention Center 10F Conference Room 1003)

Chair:Hitoshi Okazawa(Tokyo Medical and Dental University, Japan), Haruhisa Inoue(Dept. of Cell Growth and Differentiation, 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.

[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.

[NFS-02-2] Block of neuraxial degeneration by silencing an ALS-causing mutant gene with subpial AAV9 delivery

Martin Marsala1, Don W. Cleveland2, Brian K. Kaspar3, Mariana Bravo-hernandez1, Takahiro Tadokoro1 (1.University of California, San Diego, USA, 2.Ludwig Institute for Cancer Research and Department of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, USA;, 3.Avexis Inc., Chicago, USA)

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.

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

Shin-ichi Muramatsu1,2,3 (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.

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

Hitoshi Okazawa1,2 (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.

[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.