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Targeting noncoding RNAs in disease
Abstract:
Many RNA species have been identified as important players in the development of chronic diseases, including cancer. Over the past decade, numerous studies have highlighted how regulatory RNAs such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs) play crucial roles in the development of a disease state. It is clear that the aberrant expression of miRNAs promotes tumor initiation and progression, is linked with cardiac dysfunction, allows for the improper physiological response in maintaining glucose and insulin levels, and can prevent the appropriate integration of neuronal networks, resulting in neurodegenerative disorders. Because of this, there has been a major effort to therapeutically target these noncoding RNAs. In just the past 5 years, over 100 antisense oligonucleotide-based therapies have been tested in phase I clinical trials, a quarter of which have reached phase II/III. Most notable are fomivirsen and mipomersen, which have received FDA approval to treat cytomegalovirus retinitis and high blood cholesterol, respectively. The continued improvement of innovative RNA modifications and delivery entities, such as nanoparticles, will aid in the development of future RNA-based therapeutics for a broader range of chronic diseases. Here we summarize the latest promises and challenges of targeting noncoding RNAs in disease.
Insights
Regulatory RNAs, like microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), are key in chronic diseases. Targeting these noncoding RNAs with new therapies shows promise for treating various conditions.
Area of Science:
- Biomedical Science
- Molecular Biology
- Genetics
Background:
- Regulatory RNAs, including microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), are increasingly recognized for their roles in chronic diseases.
- Aberrant expression of miRNAs is implicated in cancer, cardiac dysfunction, metabolic disorders, and neurodegenerative diseases.
Purpose of the Study:
- To summarize the current landscape of targeting noncoding RNAs for therapeutic purposes.
- To highlight the recent advancements and existing challenges in developing noncoding RNA-based therapies.
Main Methods:
- Review of recent scientific literature on noncoding RNA therapeutics.
- Analysis of clinical trial data for antisense oligonucleotide-based therapies.
- Discussion of RNA modification and delivery technologies.
Main Results:
- Over 100 antisense oligonucleotide therapies have entered clinical trials in the last 5 years, with a significant portion progressing to later phases.
- FDA-approved therapies like fomivirsen and mipomersen demonstrate the clinical viability of targeting specific RNA molecules.
- Advancements in RNA modifications and delivery systems, such as nanoparticles, are crucial for future therapeutic development.
Conclusions:
- Targeting noncoding RNAs represents a promising therapeutic strategy for a wide range of chronic diseases.
- Continued innovation in RNA-based therapies is essential to overcome current challenges and expand treatment options.
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