American and South Korean researchers have studied the myocardial dysfunction observed in Duchenne muscular dystrophy. Their work involved a transgenic mouse with a combined but partial dystrophin and utrophin (mdx/utrn (+/-)) deficiency:
- an inhibitory microRNA was delivered to the animal via an AAV (adeno-associated virus) type 9 (AAV9)
- to counter the effects of miRNA25, an RNA sequence known to inhibit sarcoplasmic reticulum calcium ATPase type 2a and a Smad7-type protein,
- intravenous injection of this cardiac miR25 inhibitor was able to reduce myocardial fibrosis and improve cardiac function.
These observations could ultimately have a therapeutic impact.
