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Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations
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[RNAissance: is there a future for RNA therapeutics?]
R Bijkerk1,2, A J van Zonneveld3, E P van der Veer3
1LUMC, afd. Interne Geneeskunde, Leiden (tevens: EinthovenLaboratoryforVascularandRegenerativeMedicine).
Nederlands Tijdschrift Voor Geneeskunde
|July 3, 2020
Summary
The RNAissance revolution reveals RNA
Area of Science:
- Molecular Biology
- Genetics
- Pharmacology
Background:
- The central dogma of molecular biology traditionally describes genetic information flow from DNA to RNA to proteins.
- Recent technological advancements, termed the 'RNAissance', have uncovered crucial roles for RNA beyond protein synthesis.
- RNA molecules are now recognized for their significant involvement in various health and disease states.
Purpose of the Study:
- To highlight the expanding role of RNA in biological processes and disease.
- To underscore the growing therapeutic potential of RNA-based medicines.
- To discuss the implications of RNA therapeutics for future medical treatments.
Main Methods:
- Review of recent technological advancements in RNA research.
- Analysis of clinical trial data for RNA-based therapies.
- Examination of the pharmaceutical industry's investment in RNA therapeutics.
Main Results:
- Over 100 RNA-based therapies have entered Phase 1 clinical trials in the last five years.
- A quarter of these RNA therapies have progressed to Phase 2 or 3 trials.
- There is a significant increase in pharmaceutical companies developing RNA therapeutics.
Conclusions:
- RNA plays a vital role in both health and disease, extending beyond its traditional role in protein synthesis.
- RNA therapeutics demonstrate immense potential for treating a wide range of chronic conditions.
- Addressing toxicity and cost challenges will be key to the widespread clinical adoption of RNA-based medicines.
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