Development of an inhibitory peptide for myostatin: its implications for muscular dystrophy and sarcopenia

2016 Fiscal Year Final Research Report

• Project/Area Number: 26461285
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Neurology
• Research Institution: Kawasaki Medical School
• Principal Investigator: Yutaka Ohsawa 川崎医科大学, 医学部, 講師 (80246511)
• Co-Investigator(Kenkyū-buntansha): 西松 伸一郎 川崎医科大学, 医学部, 准教授 (20222185) ; 村上 龍文 川崎医科大学, 医学部, 准教授 (30330591)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: 筋ジストロフィー / サルコペニア / マイオスタチン / 再生医療

Outline of Final Research Achievements

Myostatin, a muscle-specific transforming growth factor-beta, negatively regulates skeletal muscle mass. The N-terminal prodomain of myostatin noncovalently binds to and suppresses the C-terminal mature domain (ligand) as an inactive circulating complex. Here, we identified the inhibitory core (IC) region that inhibited myostatin-induced transcriptional activity by 79% compared with the full-length prodomain. We found that the IC suppresses not only the ligand, but also prevents two distinct membrane receptors from binding to the ligand. Indeed, the IC inhibits myostatin, but not TGF-beta 1, and activin. Local injection of the IC peptide ameliorates muscle atrophy in a rodent model of muscular dystrophy (Ohsawa, et al. PLoS One 10:e0133713 2015,, 2015).

Free Research Field

筋ジストロフィー