期刊
JACC-BASIC TO TRANSLATIONAL SCIENCE
卷 3, 期 3, 页码 391-402出版社
ELSEVIER SCIENCE INC
DOI: 10.1016/j.jacbts.2018.03.007
关键词
cardiomyopathy; L-type calcium channels; mitochondria
资金
- Raine Priming (RPG50)
- Muscular Dystrophy Association USA [272200]
- National Health and Medical Research Foundation of Australia [1062740, 1043758]
- National Health and Medical Research Council of Australia [1062740] Funding Source: NHMRC
Current clinical trials demonstrate Duchenne muscular dystrophy (DMD) patients receiving phosphorodiamidate morpholino oligomer (PMO) therapy exhibit improved ambulation and stable pulmonary function; however, cardiac abnormalities remain. Utilizing the same PMO chemistry as current clinical trials, we have identified a non-toxic PMO treatment regimen that restores metabolic activity and prevents DMD cardiomyopathy. We propose that a treatment regimen of this nature may have the potential to significantly improve morbidity and mortality from DMD by improving ambulation, stabilizing pulmonary function, and preventing the development of cardiomyopathy. (C) 2018 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation.
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