TY - JOUR
T1 - A Morpholino Oligomer Therapy Regime That Restores Mitochondrial Function and Prevents mdx Cardiomyopathy
AU - Viola, Helena M.
AU - Johnstone, Victoria P.A.
AU - Adams, Abbie M.
AU - Fletcher, Susan
AU - Hool, Livia C.
PY - 2018/6/1
Y1 - 2018/6/1
N2 - 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.
AB - 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.
KW - cardiomyopathy
KW - L-type calcium channels
KW - mitochondria
UR - http://www.scopus.com/inward/record.url?scp=85048413211&partnerID=8YFLogxK
U2 - 10.1016/j.jacbts.2018.03.007
DO - 10.1016/j.jacbts.2018.03.007
M3 - Article
C2 - 30062225
AN - SCOPUS:85048413211
VL - 3
SP - 391
EP - 402
JO - JACC: Basic to Translational Science
JF - JACC: Basic to Translational Science
SN - 2452-302X
IS - 3
ER -