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A Morpholino Oligomer Therapy Regime That Restores Mitochondrial Function and Prevents mdx Cardiomyopathy
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,...
Autores principales: | Viola, Helena M., Johnstone, Victoria P.A., Adams, Abbie M., Fletcher, Susan, Hool, Livia C. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6059013/ https://www.ncbi.nlm.nih.gov/pubmed/30062225 http://dx.doi.org/10.1016/j.jacbts.2018.03.007 |
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