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

作者：Viola, Helena M.; Johnstone, Victoria P. A.; Adams, Abbie M.; Fletcher, Susan; Hool, Livia C.^*

来源：JACC-Basic to Translational Science, 2018, 3(3): 391-402.

DOI：10.1016/j.jacbts.2018.03.007

• cardiomyopathy
• L-type calcium channels
• mitochondria

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.

• 出版日期2018-6