A morpholino oligomer therapy regime that restores mitochondrial function and prevents mdx cardiomyopathy

Viola, H.M., Johnstone, V.P.A., Adams, A.M., Fletcher, S. and Hool, L.C. (2018) A morpholino oligomer therapy regime that restores mitochondrial function and prevents mdx cardiomyopathy. JACC: Basic to Translational Science, 3 (3). pp. 391-402.

Abstract

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.

Item Type:	Journal Article
Murdoch Affiliation(s):	Centre for Comparative Genomics
Publisher:	Elsevier on behalf of the American College of Cardiology Foundation
Copyright:	© 2018 The Authors.
URI:	http://researchrepository.murdoch.edu.au/id/eprint/41268

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