Emerging Concepts in miRNA-Based Therapeutics

Dear Colleagues,

microRNAs (miRNAs, miRs) are short (18–26 nucleotides) regulatory RNA molecules. They were discovered in 1993 in Caenorhabditis elegans (C. elegans). Since then, there has been an exponential growth in the number of publications describing new members of this emergent nucleic acid family, elucidating their biogenesis, mechanisms of action, and their roles in diverse signaling pathways and physiological processes, as well as their contribution to the development of multiple pathologies.

miRNAs act mainly by downregulating genes to whose mRNAs they are (partially) complementary. As almost ubiquitous master regulators of crucially important cellular functions and physiological processes, in recent years, miRNAs have become new promising diagnostic tools and therapeutic targets in multiple pathological conditions, from asthma and cancer, through cardiovascular and metabolic diseases, to Parkinson’s disease and Zika virus infection.

Some therapeutic applications reached clinical trial stages, but in spite of great progress in basic research and preclinical studies and the development of stable and effective delivery vehicles, as of today, no miRNA-based therapeutic has been approved for treatment. However, there is a consensus in the gene therapy field that this is just a matter of time.

Some of the main obstacles in fulfilling miRNAs’ promise as therapeutic agents are the wide range of downstream effects and safe and effective targeted delivery.

This Special Issue aims at presenting the most recent advancements in miRNA-based therapeutics, including new miRNA targets and novel promising approaches and solutions to overcoming existing obstacles in therapeutic applications. It also invites new perspectives and paradigms that might lead to a breakthrough in the field, hopefully leading to the development of the first approved miRNA-based therapeutic agents.

Dr. Anetta Wronska
Guest Editor

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• microRNA
• miRNA
• miR
• gene therapy
• non-coding RNA
• microRNA-based therapeutics