MicroRNAs (miRNAs) are very powerful genetic regulators, as evidenced by the
fact that a single miRNA can direct entire cellular pathways via interacting with
a broad spectrum of target genes. This property renders miRNAs as highly interesting therapeutic tools to restore cell functions that are altered as part of a disease phenotype. However, this strength of miRNAs is also a weakness because
their cellular effects are so numerous that off-target effects can hardly be
avoided. In this review, we point out the main challenges and the strategies to
specifically address the problems that need to be surmounted in the push toward
a therapeutic application of miRNAs. Particular emphasis is given to approaches
that have already found their way into clinical studies
