Abstract
RNA is the messenger molecule that conveys information from the genome and allows the production of biomolecules required for life in a responsive and regulated way. Most genes are able to produce multiple mRNA products in response to different internal or external environmental signals, in different tissues and organs, and at specific times in development or later life. This fine tuning of gene expression is dependent on the coordinated effects of a large and intricate set of regulatory machinery, which together orchestrate the genomic output at each locus and ensure that each gene is expressed at the right amount, at the right time and in the correct location. This complexity of control, and the requirement for both sequence elements and the entities that bind them, results in multiple points at which errors may occur. Errors of RNA biology are common and found in association with both rare, single gene disorders, but also more common, chronic diseases. Fortunately, complexity also brings opportunity. The existence of many regulatory steps also offers multiple levels of potential therapeutic intervention which can be exploited. In this review, I will outline the specific points at which coding RNAs may be regulated, indicate potential means of intervention at each stage, and outline with examples some of the progress that has been made in this area. Finally, I will outline some of the remaining challenges with the delivery of RNA-based therapeutics but indicate why there are reasons for optimism.
Highlights
Specialty section: This article was submitted to RNA, a section of the journalFrontiers in GeneticsReceived: 18 January 2019 Accepted: 26 February 2019Published: 08 March 2019Citation: Harries LW (2019) RNA Biology Provides New Therapeutic Targets for Human Disease
There have been a number of examples, where the specifics of RNA regulatory machinery have been harnessed to produce novel therapeutics that are in phase III clinical trials [e.g., Patisiran for Familial amyloid polyneuropathy (Rizk and Tuzmen, 2017), Custirsen for RNA Therapeutics for Human Disease prostate cancer (Edwards et al, 2017) and AGS-003 for renal cell carcinoma (Figlin, 2015)]
This review aims to explore the potential for intervention in messenger RNA (mRNA) processing or posttranscriptional regulation with selected examples for future therapeutic benefit
Summary
Most genes are able to produce multiple mRNA products in response to different internal or external environmental signals, in different tissues and organs, and at specific times in development or later life. This fine tuning of gene expression is dependent on the coordinated effects of a large and intricate set of regulatory machinery, which together orchestrate the genomic output at each locus and ensure that each gene is expressed at the right amount, at the right time and in the correct location.
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