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Related Concept Videos

RNA Splicing01:32

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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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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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The basic structure of RNA consists of a five-carbon sugar and one of four nitrogenous bases. Although most RNA is single-stranded, it can form complex secondary and tertiary structures. Such structures play essential roles in the regulation of transcription and translation.
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Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
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Transfer RNA Synthesis02:36

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One of the unique features of tRNA is the presence of modified bases. In some tRNAs, modified bases account for nearly 20% of the total bases in the molecule. Altogether, these unusual bases protect the tRNA from enzymatic degradation by RNases.
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Related Experiment Video

Updated: Jan 21, 2026

Method for Identifying Small Molecule Inhibitors of the Protein-protein Interaction Between HCN1 and TRIP8b
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Methods to identify and optimize small molecules interacting with RNA (SMIRNAs).

Andrei Ursu1, Simon Vézina-Dawod1, Matthew D Disney1

  • 1Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.

Drug Discovery Today
|July 30, 2019
PubMed
Summary

Small molecules interacting with RNA (SMIRNAs) are crucial for treating diseases caused by noncoding RNAs (ncRNAs). This review details methods for discovering and optimizing SMIRNAs to target ncRNAs effectively for therapeutic benefit.

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Drug Discovery

Background:

  • Noncoding RNAs (ncRNAs) play critical roles in human diseases.
  • Aberrant RNA structures and expression contribute to disease pathogenesis.
  • ncRNAs represent promising therapeutic targets.

Purpose of the Study:

  • To review methodologies for discovering and optimizing small molecules interacting with RNA (SMIRNAs).
  • To highlight the importance of evaluating direct target engagement and phenotypic rescue for SMIRNAs.
  • To advance the understanding of RNA-targeted drug development.

Main Methods:

  • Description of methodologies for discovering SMIRNAs.
  • Explanation of techniques for optimizing SMIRNAs.
  • Evaluation of methods for assessing SMIRNA efficacy in vitro and in vivo.

Main Results:

  • Identification of key strategies for SMIRNA discovery and optimization.
  • Emphasis on the necessity of validating target engagement and functional effects.
  • Discussion of approaches to characterize the mode of action of SMIRNAs.

Conclusions:

  • SMIRNAs offer a promising therapeutic avenue for ncRNA-mediated diseases.
  • Robust methodologies are essential for the rational development of RNA-targeted drugs.
  • Further research into SMIRNA characterization will accelerate their clinical translation.