The potential of exosomes for the diagnosis and treatment of Duchenne muscular dystrophy

Abstract

Duchenne muscular dystrophy is the most common type of muscular dystrophy. There is no effective cure for this disease. Recently, researchers have started to look at the therapeutic potential of exosomes — small (40–100 nm) vesicles secreted by cells into the extracellular environment. They transport a few types of macromolecules, including microRNA and proteins, that can be analyzed to estimate the efficacy of the applied therapy. Besides, exosomes can be harnessed for delivering therapeutic components (microRNA, antisense oligonucleotides) to the target tissue. Below, we analyze the available literature and assess the feasibility of using exosomes in the diagnosis and treatment of Duchenne muscular dystrophy. We conclude that exosomes can have their place in the arsenal of researchers and clinicians once some technical issues are solved.