The functional involvement of microRNAs in human neoplasia has raised in the last years an increasing interest in the scientific community toward the potential application in clinics as therapeutic tools. Indeed, the possibility to modulate their expression to re-establish a lost equilibrium and counteract tumor growth and dissemination, and/or to improve responsiveness to standard therapies, is promising and fascinating. However, several issues need to be taken into account such as factors related to miRNA stability in the blood, tissue penetration and potential off-target effects, which might affect safety, tolerability and efficacy of an miRNA-based therapy. Here we describe the most relevant challenges related to miRNA-based therapy, review the delivery strategies exploited to date and the on-going clinical trials.
Keywords: cancer; microRNA; therapy.