Citation:
JACC Basic Transl Sci. 2018 Jun 25;3(3):391-402. doi: 10.1016/j.jacbts.2018.03.007. eCollection 2018 Jun
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.
Epub:
Not Epub
Link to Publication:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6059013/
Organism or Cell Type:
mice mdx
Delivery Method:
subcutaneous injection