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Next RNA Therapeutics: The Mine of Non-Coding
Sabrina Garbo1, Rossella Maione1, Marco Tripodi1,2
1Department of Molecular Medicine, Faculty of Pharmacy and Medicine, Sapienza University of Rome, Viale Regina Elena 324, 00161 Rome, Italy.
Non-coding RNAs (ncRNAs) play crucial roles in gene regulation and disease. This review explores therapeutic ncRNA strategies, their clinical applications, and delivery challenges for treating various diseases.
Area of Science:
- Molecular Biology
- Genetics
- Biochemistry
Background:
- Non-coding RNAs (ncRNAs) are increasingly recognized for their diverse biological functions beyond protein-coding.
- ncRNAs regulate gene expression at transcriptional and post-transcriptional levels, influencing cellular processes.
- Dysregulation of ncRNAs is implicated in the onset and progression of various diseases.
Purpose of the Study:
- To review principal classes of therapeutic ncRNA molecules.
- To summarize their roles in disease onset and progression.
- To highlight clinical applications and delivery strategies for RNA therapeutics.
Main Methods:
- Literature review of ncRNA research and clinical trials.
- Analysis of ncRNA therapeutic strategies and their mechanisms.
- Discussion of advantages, limitations, and delivery system development.
Main Results:
- ncRNAs are critical regulators of gene expression and pathophysiological processes.
- Several ncRNA-based therapies are in clinical trials or use for treating diseases.
- Targeted delivery strategies are essential to enhance efficacy and minimize off-target effects.
Conclusions:
- Therapeutic ncRNAs offer promising avenues for disease treatment.
- Further development of targeted delivery systems is crucial for successful clinical translation.
- Understanding ncRNA functions is key to advancing RNA-based medicine.
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