Abstract
The first therapeutic nucleic acid, a DNA oligonucleotide, was approved for clinical use in 1998. Twenty years later, in 2018, the first therapeutic RNA-based oligonucleotide was United States Food and Drug Administration (FDA) approved. This promises to be a rapidly expanding market, as many emerging biopharmaceutical companies are developing RNA interference (RNAi)-based, and RNA-based antisense oligonucleotide therapies. However, miRNA therapeutics are noticeably absent. miRNAs are regulatory RNAs that regulate gene expression. In disease states, the expression of many miRNAs is measurably altered. The potential of miRNAs as therapies and therapeutic targets has long been discussed and in the context of a wide variety of infections and diseases. Despite the great number of studies identifying miRNAs as potential therapeutic targets, only a handful of miRNA-targeting drugs (mimics or inhibitors) have entered clinical trials. In this review, we will discuss whether the investment in finding potential miRNA therapeutic targets has yielded feasible and practicable results, the benefits and obstacles of miRNAs as therapeutic targets, and the potential future of the field.
Highlights
The majority of RNA-based drugs used in the clinic or currently in development are antisense oligonucleotides (ASOs) or doubled-stranded RNA molecules called short interfering RNAs
RNA-based drugs are the latest frontier in nucleic acid therapeutics, with a considerable number in clinical trials
This demonstrates the significant potential of these therapeutic strategies, which promise to be effective in a wide range of currently untreatable disorders
Summary
The majority of human diseases are influenced by genetic factors [1]. The first approved human gene therapy treatment was approved in 1990 to treat adenosine deaminase deficiency [2]. The advantage of gene therapy is that it can successfully cure genetic diseases as demonstrated in 2002, with patients with severe combined immunodeficiency (SCID) [3]. Gene therapies for more complex disorders like neurodegenerative Alzheimer’s disease [4] and polygenic cancers are in development [5]. DNA-based therapeutics, are not restricted to gene replacement [6]. DNA vaccines are currently restricted to veterinary clinics. The first nucleic acid drug to get United States Food and Drug Administration (FDA) approval was fomivirsen (Vitravene), developed for immunocompromised patients suffering with cytomegalovirus (CMV) retinitis [8]. Many biopharmaceutical companies are focusing on another nucleic acid for its therapeutic potential—RNA
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