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Related Concept Videos

Riboswitches01:56

Riboswitches

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.
The aptamer has high specificity for a particular metabolite which allows riboswitches to specifically regulate...
Regulated mRNA Transport02:22

Regulated mRNA Transport

In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing specific...
Regulated mRNA Transport02:22

Regulated mRNA Transport

In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing specific...
Improving Translational Accuracy02:07

Improving Translational Accuracy

Base complementarity between the three base pairs of mRNA codon and the tRNA anticodon is not a failsafe mechanism. Inaccuracies can range from a single mismatch to no correct base pairing at all. The free energy difference between the correct and nearly correct base pairs can be as small as 3 kcal/ mol. With complementarity being the only proofreading step, the estimated error frequency would be one wrong amino acid in every 100 amino acids incorporated. However, error frequencies observed in...
Regulation of Expression Occurs at Multiple Steps02:24

Regulation of Expression Occurs at Multiple Steps

Gene expression can be regulated at almost every step from gene to protein. Transcription is the step that is most commonly regulated. This involves the binding of proteins to short regulatory sequences on the DNA. This association can either promote or inhibit the transcription of a gene associated with the respective sequence.
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
Ribosome Profiling02:24

Ribosome Profiling

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
Ribosome profiling has many applications, including in vivo monitoring of translation inside a particular organ or tissue type and quantifying new protein synthesis levels.
The technique helps...

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Related Experiment Video

Updated: Jun 5, 2026

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
09:44

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes

Published on: March 3, 2015

Towards mRNA therapeutics 2.0.

Kenneth R Chien1, Kylie S Foo2,3, Nevin Witman2,4

  • 1Department of Cell and Molecular Biology, Karolinska Institutet, Stockholm, Sweden. Kenneth.chien@ki.se.

Nature Reviews. Drug Discovery
|June 3, 2026
PubMed
Summary
This summary is machine-generated.

Messenger RNA (mRNA) therapeutics show promise beyond vaccines, offering new treatments for rare and common diseases. Advances in delivery and formulation are overcoming barriers to unlock mRNA drug potential.

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Last Updated: Jun 5, 2026

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Area of Science:

  • Biotechnology
  • Genetics
  • Immunology

Background:

  • Messenger RNA (mRNA) technology has revolutionized vaccine development.
  • mRNA also presents significant therapeutic potential for various diseases.
  • Clinical trials are identifying barriers to mRNA drug development.

Purpose of the Study:

  • To review scientific insights from clinical studies on mRNA-based drugs.
  • To highlight advances addressing key barriers in mRNA therapeutics.
  • To explore the integration of mRNA therapy with gene and cell therapies.

Main Methods:

  • Review of select clinical studies on mRNA-based drugs.
  • Analysis of interdisciplinary technical advances in mRNA drug development.
  • Examination of innovative approaches for mRNA delivery and formulation.

Main Results:

  • mRNA therapies are being explored for enzyme replacement, cancer immunotherapy, genome modification, and immune cell reprogramming.
  • Key barriers include delivery systems, immune responses to vehicles, and organ-specific targeting.
  • Novel approaches are being developed to enable in vivo delivery, immune-silent formulations, and non-liver targeting.

Conclusions:

  • mRNA therapeutics have broad potential for rare and common diseases.
  • Overcoming delivery and immunogenicity challenges is crucial for advancing mRNA drugs.
  • Interdisciplinary innovations are paving the way for next-generation mRNA therapeutics (therapeutics 2.0).