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2017 Fiscal Year Final Research Report

development of a new remedy for muscular dystrophies

Research Project

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Project/Area Number 15K08312
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Pathological medical chemistry
Research InstitutionSapporo Medical University

Principal Investigator

Horio Yoshiyuki  札幌医科大学, 医学部, 教授 (30181530)

Co-Investigator(Kenkyū-buntansha) 浜田 博喜  岡山理科大学, 理学部, 教授 (10164914)
Co-Investigator(Renkei-kenkyūsha) NIKAIDO Koki  札幌医科大学, 医学部, 小児科学講座 (90359993)
Project Period (FY) 2015-04-01 – 2018-03-31
KeywordsSIRT1 / レスベラトロール / 筋ジストロフィー / オートファジー / マイトファジー / 活性酸素 / 臨床研究
Outline of Final Research Achievements

There exists few remedy to muscular dystrophies (MD) except glucocorticoids, but there is no report of glucocorticoids for extending walking ability more than 2 years. We found that resveratrol, an activator of SIRT1, is effective on dystrophin-deficient mdx mice.
In the present study, we found that autophagy of damaged mitochondria containing deleted mitochondria DNA was suppressed in mdx mice. Damaged mitochondria liberate high levels of reactive oxygen species (ROS). Resveratrol reactivated the autophagy, which cleared damaged mitochondria and reduced ROS. Administration of resveratrol to 11 MD patients for 24 weeks showed resveratrol was effective on MD. Resveratrol significantly increased motor function and enhanced muscle strength with quantitative muscle testing. Thus, resveratrol will be a promising remedy for MD.

Free Research Field

病態生化学

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Published: 2019-03-29  

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