Welcome to Department of Molecular Therapy, a major basic-science department at National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo.

Our department is dedicated to elucidating the molecular pathogenesis of neuromuscular disorders, especially muscular dystrophy, and the development of advanced gene- and stem cell-based therapeutics for these diseases. For this purpose, we integrate molecular, pharmacologic, proteomic, and genomic methodologies using various types of unique animal models, including dystrophic dogs, mice, rats, and common marmosets. We are combating these diseases through research aimed at implementing promising novel therapeutic strategies, including oligonucleotide-based exon-skipping therapy and induced pluripotent stem cell (iPSC)-based cell therapy, into clinical practice. One of the significant achievements of our department is the approval of our antisense oligonucleotide-based exon 53-skipping drug, viltolarsen (NS-065/NCNP-01), for muscular dystrophy in Japan, following a successful investigator-initiated trial of NS-065/NCNP-01 (Sci Transl Med. 2018;10).

Topics

eSkip-Finder is launched ⇒　 https://eskip-finder.org

It is a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping.

https://www.ncnp.go.jp/topics/2021/20210609p.html

Marketing authorization of NS-065/NCNP-01, viltolarsen, for the treatment of Duchenne muscular dystrophy

Viltolarsen (VILTEPSO^® Intravenous Infusion 250 mg) has been approved in Japan for the treatment of patients with Duchenne amenable to exon 53 skipping therapy. The drug was granted priority review in the USA with a decision expected in the third quarter of 2020.

https://www.amed.go.jp/en/news/release_20200327.html

News

• On 1st September 2021,

  Updated Members.

• On 1st August 2021,

  Updated Publications.

• On 1st August 2021,

  Updated column.

• On 1st April 2021,

  Updated Publications.

• On 1st April 2021,

  Updated Members.

• On 1st April 2021,

  Updated column.

• On 18th December 2020,

  Updated column.

• On 22th October 2020,

  Updated Work or study with us !

• On 6th October 2020,

  Updated column.

• On 24th September 2020,

  The top 1% article of the academic field of Clinical Medicine based on a highly cited threshold for the field and publication year (Web of Science) : A Review of Duchenne Muscular Dystrophy by Dr. Y. Shimizu-Motohashi. J. Pers. Med. 2019, 9(1), 1

• On 17th September 2020,

  Dr. Hotake Takizawa, a Researcher at Department of Molecular Therapy, received the Best Oral Award in Clinical Category at the 61st Annual Meeting of the Japanese Society of Neurology.

• On 24th August 2020, our website has been totally renewed.

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