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JST Press Release

December 15, 2014
Japan Science and Technology Agency (JST)
5-3, Yonbancho, Chiyoda-ku, Tokyo 102-8666

Gene therapy of model mice for spinocerebellar ataxia was successful.
- Expectation for therapeutic development of neurodegenerative diseases -

AAV vector-mediated complementation of HMGB1, which possesses a profound effect on DNA-mediated cellular functions, successfully elongated the lifespan and improved the motor activity of model mice for spinocerebellar ataxia type 1(SCA1). Especially, the extent of lifespan elongation was the top result in the field. In addition this study revealed the involvement of HMGB1 in mitochondrial DNA damage repair.

Researcher Information

Research Area “Creation of a Novel Technology towards Diagnosis and Treatment Based on Understanding of Molecular Pathogenesis of Psychiatric and Neurological Disorders”
Research Theme “Development of Comprehensive Therapies for Polyglutamine Diseases”

Journal Information

Hikaru Ito, Kyota Fujita, Kazuhiko Tagawa, Xigui Chen, Hidenori Homma, Toshikazu Sasabe, Jun Shimizu, Shigeomi Shimizu, Takuya Tamura, Shin-ichi Muramatsu, and Hitoshi Okazawa. “HMGB1 facilitates repair of mitochondrial DNA damage and extends the lifespan of mutant ataxin-1 knock-in mice”. EMBO Molecular Medicine, Published online 15.12.2014, doi: 10.15252/emmm.201404392.


[About Research]
Hitoshi Okazawa, M.D., Ph.D.
Professor, Department of Neuropathology, Division of Pathophysiology, Medical Research Institute, Tokyo Medical and Dental University

[About Program]
Takashi Kawaguchi
Life Innovation Group, Department of Innovation Research, JST
URL: http://www.jst.go.jp/kisoken/crest/en/index.html


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