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RNA Therapeutics: How Far Have We Gone?
Maria Francisca Coutinho1, Liliana Matos1, Juliana Inês Santos1
1Department of Human Genetics, National Institute of Health Doutor Ricardo Jorge, Porto, Portugal.
Advances in Experimental Medicine and Biology
|July 26, 2019
Summary
RNA therapeutics show great promise, targeting diseases at DNA, mRNA, or pre-mRNA levels. This review covers current RNA-based strategies, clinical applications, and challenges in their development.
Area of Science:
- Biotechnology
- Molecular Biology
- Therapeutics Development
Background:
- RNA molecules are emerging as key targets for novel therapeutic interventions.
- Numerous RNA-based therapies are under investigation in preclinical and clinical settings, with some already approved.
- RNA therapeutics offer diverse mechanisms to modulate gene expression and correct genetic defects.
Purpose of the Study:
- To review various RNA-based therapeutic approaches.
- To discuss their translation into clinical practice.
- To highlight challenges and ongoing research to overcome them.
Main Methods:
- Review of current literature on RNA-based therapeutic strategies.
- Analysis of clinical trial data and approved RNA-based treatments.
- Discussion of different RNA targeting levels: pre-mRNA, mRNA, and DNA.
Main Results:
- RNA therapeutics can target pre-mRNA (splicing modulation), mRNA (gene expression inhibition), and DNA (gene editing).
- Approaches include antisense oligonucleotides, siRNAs, U1snRNA vectors, CRISPR/Cas systems, in vitro transcribed mRNA, and aptamers.
- Significant progress has been made in translating these approaches to clinical applications.
Conclusions:
- RNA-based therapeutics represent a rapidly advancing field with broad clinical potential.
- Overcoming delivery, stability, and off-target effects are key challenges.
- Continued research is crucial for optimizing RNA therapeutic efficacy and safety.
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