Abstract
New technologies have greatly shaped the scientific and medical landscape within the last years. The unprecedented expansion of data and information on RNA biology has led to the discovery of new RNA classes with unique functions and unexpected modifications. Today, the biggest challenge is to transfer the large number of findings in basic RNA biology into corresponding clinical RNA-based therapeutics. Lately, this research begins to yield positive outcomes. RNA drugs advance to the final phases of clinical trials or even receive FDA approval. Furthermore, the introduction of the RNA-guided gene-editing technology CRISPR and advances in the delivery of messenger RNAs have triggered a major progression in the field of RNA-therapeutics. Especially short interfering RNAs and antisense oligonucleotides are promising examples for novel categories of therapeutics. However, several issues need to be addressed including intracellular delivery, toxicity, and immune responses before utilizing RNAs in a clinical setting. In this review, we provide an overview on opportunities and challenges for clinical translation of RNA-based therapeutics, with an emphasis on advances in novel delivery technologies and abdominal aortic aneurysm disease where non-coding RNAs have been shown to play a crucial regulatory role.
Highlights
Abdominal aortic aneurysm (AAA) is a common disease and defined as an abnormal focal dilation of the infrarenal aorta over 50% of the normal diameter [1]
Perfect basepair matching between the short interfering RNA (siRNA) strand and the target mRNA is required for cleavage and subsequent degradation, while imperfect base-pair matching can result in miR-like repression [19, 21]
On the opposite site of the non-coding RNA spectrum, we find so called long non-coding RNAs [27]
Summary
We believe that through the hereby provided review, we can give the interested readership a thoughtfully cured overview regarding the following topics: 1. A broad review on the ever-growing spectrum of RNAs with a focus on non-coding RNAs (including small and long non-coding RNAs). We believe that through the hereby provided review, we can give the interested readership a thoughtfully cured overview regarding the following topics: 1. A broad review on the ever-growing spectrum of RNAs with a focus on non-coding RNAs (including small and long non-coding RNAs). 2. An introduction to a widespread range of translational methods to apply RNAs as therapeutics (including nanocarriers and extracellular vesicles). 3. A comprehensive insight into challenges of translation in the field of RNAs. 4. Instructive examples where the mentioned challenges have been accepted and first results have been reached (including atherosclerosis and aneurysm disease)
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