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Related Concept Videos

Experimental RNAi02:15

Experimental RNAi

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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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RNA-seq03:21

RNA-seq

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RNA sequencing, or RNA-Seq, is a high-throughput sequencing technology used to study the transcriptome of a cell. Transcriptomics helps to interpret the functional elements of a genome and identify the molecular constituents of an organism. Additionally, it also helps in understanding the development of an organism and the occurrence of diseases. 
Before the discovery of RNA-seq, microarray-based methods and Sanger sequencing were used for transcriptome analysis. However, while...
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Riboswitches01:56

Riboswitches

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Riboswitches are non-coding mRNA domains that regulate the transcription and translation of downstream genes without the help of proteins. Riboswitches bind directly to a metabolite and can form unique stem-loop or hairpin structures in response to the amount of the metabolite present. They have two distinct regions – a metabolite-binding aptamer and an expression platform.
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Leaky Scanning02:28

Leaky Scanning

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During most eukaryotic translation processes, the small 40S ribosome subunit scans an mRNA from its 5' end until it encounters the first start AUG codon. The large 60S ribosomal subunit then joins the smaller one to initiate protein synthesis. The location of the translation initiation is largely determined by the nucleotides near the start codon as there may be multiple translation initiation sites present on the mRNA.  Marilyn Kozak discovered that the sequence RCCAUGG (where R...
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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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Ribosome Profiling02:24

Ribosome Profiling

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Ribosome profiling or ribo-sequencing is a deep sequencing technique that produces a snapshot of active translation in a cell. It selectively sequences the mRNAs protected by ribosomes to get an insight into a cell’s translation landscape at any given point in time.
Applications of ribosome profiling
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Updated: Jun 26, 2025

Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations
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Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations

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Discovery platforms for RNA therapeutics.

Giulio Ciucci1, Luca Braga2, Serena Zacchigna1,3

  • 1Cardiovascular Biology Laboratory, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.

British Journal of Pharmacology
|May 18, 2024
PubMed
Summary
This summary is machine-generated.

This review details technological platforms for discovering RNA therapeutics, focusing on those reaching advanced clinical stages or market approval. It highlights methods for targeting previously undruggable diseases.

Keywords:
RNA therapeuticsdrug discoveryfunctional screening

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Area of Science:

  • Biotechnology and Pharmaceutical Sciences
  • Molecular Biology and Genetics

Background:

  • RNA therapeutics offer novel strategies for diseases previously considered undruggable.
  • Existing reviews extensively cover RNA therapeutic advantages and delivery, but lack focus on discovery platforms.

Approach:

  • This article surveys current ex vivo assays and technological platforms used for RNA therapeutic discovery.
  • It analyzes the pros and cons of each platform and their applications.

Key Points:

  • Focus on platforms relevant to RNA therapies in Phase 3 trials or approved.
  • Discussion includes advantages, disadvantages, and application areas of discovery technologies.

Conclusions:

  • Technological platforms are crucial for advancing RNA therapeutics from discovery to clinical application.
  • Understanding these platforms is key to developing new treatments for challenging diseases.