Cancer and organ injury-such as that occurring in the
perioperative period, including
acute lung injury,
myocardial infarction, and acute gut injury-are among the leading causes of death in the United States and impose a significant impact on quality of life.
MicroRNAs (
miRNAs) have been studied extensively during the last two decades for their role as regulators of gene expression, their translational application as diagnostic markers, and their potential as therapeutic targets for disease treatment. Despite promising preclinical outcomes implicating
miRNA targets in disease treatment, only a few
miRNAs have reached clinical trials. This likely relates to difficulties in the delivery of
miRNA drugs to their targets to achieve efficient inhibition or overexpression. Therefore, understanding how to efficiently deliver
miRNAs into diseased tissues and specific cell types in patients is critical. This review summarizes current knowledge on various approaches to deliver therapeutic
miRNAs or
miRNA inhibitors and highlights current progress in
miRNA-based disease
therapy that has reached clinical trials. Based on ongoing advances in
miRNA delivery, we believe that additional therapeutic approaches to modulate
miRNA function will soon enter routine medical treatment of human disease, particularly for
cancer or perioperative organ injury. SIGNIFICANCE STATEMENT:
MicroRNAs have been studied extensively during the last two decades in
cancer and organ injury, including
acute lung injury,
myocardial infarction, and acute gut injury, for their regulation of gene expression, application as diagnostic markers, and therapeutic potentials. In this review, we specifically emphasize the pros and cons of different delivery approaches to modulate
microRNAs, as well as the most recent exciting progress in the field of therapeutic targeting of
microRNAs for disease treatment in patients.