2013 Fiscal Year Final Research Report

Establishment of a new therapeutic protocol for cardiomyopathy in Duchenne muscular dystrophy.

Research Project

Project/Area Number	24791059
Research Category	Grant-in-Aid for Young Scientists (B)
Allocation Type	Multi-year Fund
Research Field	Pediatrics
Research Institution	Kyoto University
Principal Investigator	BABA Shiro 京都大学, 医学(系)研究科(研究院), 助教 (60432382)
Project Period (FY)	2012-04-01 – 2014-03-31
Keywords	デュシェンヌ型筋ジストロフィー / 心筋症 / iPS細胞 / カルシウム代謝
Research Abstract	Duchenne muscular dystrophy (DMD) frequently causes cardiomyopathy. Because of development of home respirator and oxygen treatment, a death rate by respiratory failure has been remarkably reduced and the patients live longer than before. In contrast, the death rate by heart failure is increasing. To reveal mechanisms of developing cardiomyopathy in DMD patients, we generated iPSC from a DMD patient and differentiated cardiomyocytes from the iPS cells. When we measured intracellular free calcium ion concentration, calcium level was remarkably elevated in the cardiomyocytes derived from DMD-iPS cardiomyocytes. Moreover, calcium overload was observed much more clearly in cardiomyocytes derived from DMD-iPSCs than in cardiomyocytes derived from control-hiPSCs after compulsory stretching.These findings indicated that calcium overload is one of the mechanisms for cardiomyopathy in DMD patients. Reducing calcium overload will be one of the treatments for DMD cardiomyopathy patients.