Viola, Helena M and Johnstone, Victoria P A and Adams, Abbie M and Fletcher, Susan and Hool, Livia 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. ISSN 2452302X (PMC OA)
Full text not available from this repository.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: | Article |
|---|---|
| Additional Information: | This article is available for free from the PMC website: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6059013/ |
| Subjects: | R Medicine > R Medicine (General) |
| Depositing User: | Repository Administrator |
| Date Deposited: | 05 Aug 2018 23:47 |
| Last Modified: | 05 Aug 2018 23:54 |
| URI: | https://eprints.victorchang.edu.au/id/eprint/748 |
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