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

H.M. Viola; V.P.A. Johnstone; A.M. Adams; S. Fletcher; L.C. Hool

doi:10.1016/j.jacbts.2018.03.007

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

Journal article

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

H.M. Viola, V.P.A. Johnstone, A.M. Adams, S. Fletcher and L.C. Hool

JACC: Basic to Translational Science, Vol.3(3), pp.391-402

2018

DOI: https://doi.org/10.1016/j.jacbts.2018.03.007

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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.

Details

Title: A morpholino oligomer therapy regime that restores mitochondrial function and prevents mdx cardiomyopathy
Authors/Creators: H.M. Viola (Author/Creator) - The University of Western Australia
V.P.A. Johnstone (Author/Creator) - The University of Western Australia
A.M. Adams (Author/Creator) - Murdoch University
S. Fletcher (Author/Creator) - Murdoch University
L.C. Hool (Author/Creator) - The University of Western Australia
Publication Details: JACC: Basic to Translational Science, Vol.3(3), pp.391-402
Publisher: Elsevier on behalf of the American College of Cardiology Foundation
Identifiers: 991005541080807891
Copyright: © 2018 The Authors.
Murdoch Affiliation: Centre for Comparative Genomics
Language: English
Resource Type: Journal article

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Collaboration types: Domestic collaboration
Citation topics: 1 Clinical & Life Sciences; 1.255 Musculoskeletal Disorders; 1.255.628 Duchenne Muscular Dystrophy
Web Of Science research areas: Cardiac & Cardiovascular Systems
ESI research areas: Clinical Medicine