A Morpholino Oligomer Therapy Regime That Restores Mitochondrial Function and Prevents mdx Cardiomyopathy

Helena M. Viola, Victoria P.A. Johnstone, Abbie M. Adams, Susan Fletcher, Livia C. Hool

Research output: Contribution to journalArticlepeer-review

8 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)391-402
Number of pages12
JournalJACC: Basic to Translational Science
Volume3
Issue number3
DOIs
Publication statusPublished - 1 Jun 2018

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