Title |
A Morpholino Oligomer Therapy Regime That Restores Mitochondrial Function and Prevents mdx Cardiomyopathy
|
---|---|
Published in |
JACC: Basic to Translational Science, June 2018
|
DOI | 10.1016/j.jacbts.2018.03.007 |
Pubmed ID | |
Authors |
Helena M. Viola, Victoria P.A. Johnstone, Abbie M. Adams, Susan Fletcher, Livia C. Hool |
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. |
X Demographics
Geographical breakdown
Country | Count | As % |
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United States | 7 | 27% |
Italy | 3 | 12% |
Spain | 2 | 8% |
Chile | 2 | 8% |
Australia | 1 | 4% |
United Kingdom | 1 | 4% |
Unknown | 10 | 38% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 15 | 58% |
Scientists | 7 | 27% |
Practitioners (doctors, other healthcare professionals) | 2 | 8% |
Science communicators (journalists, bloggers, editors) | 2 | 8% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 11 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Bachelor | 3 | 27% |
Other | 1 | 9% |
Professor | 1 | 9% |
Student > Ph. D. Student | 1 | 9% |
Researcher | 1 | 9% |
Other | 1 | 9% |
Unknown | 3 | 27% |
Readers by discipline | Count | As % |
---|---|---|
Agricultural and Biological Sciences | 4 | 36% |
Medicine and Dentistry | 2 | 18% |
Chemistry | 1 | 9% |
Neuroscience | 1 | 9% |
Unknown | 3 | 27% |