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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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Intact DNA strands can be found in fossils, while scientists sometimes struggle to keep RNA intact under laboratory conditions. The structural variations between RNA and DNA underlie the differences in their stability and longevity. Because DNA is double-stranded, it is inherently more stable. The single-stranded structure of RNA is less stable but also more flexible and can form weak internal bonds. Additionally, most RNAs in the cell are relatively short, while DNA can be up to 250 million...
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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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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

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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.