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Related Concept Videos

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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Translational regulation in prokaryotes ensures efficient protein synthesis by controlling ribosome access to mRNA. This regulation is mediated by secondary RNA structures, including translational riboswitches, RNA thermometers, and small RNAs (sRNAs), which respond to intracellular and environmental signals to modulate gene expression.Translational RiboswitchesRiboswitches in the leader region of mRNAs can regulate translation by altering the accessibility of the Shine-Dalgarno (SD) sequence,...
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Cardiovascular diseases, encompassing a range of conditions, can significantly affect the heart's operations and the overall circulatory system. These conditions impair the heart's ability to pump blood, leading to a deficit in oxygen supply to crucial organs. Anomalies in the heart's electrical system, known as arrhythmias, can cause heartbeats to accelerate or slow down. Usually, heart rates increase during physical activity and decrease while resting or sleeping. However,...
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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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Updated: Aug 28, 2025

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model
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Small non-coding RNA therapeutics for cardiovascular disease.

Ajay M Shah1, Mauro Giacca1

  • 1King's College London, British Heart Foundation Centre of Research Excellence, School of Cardiovascular and Metabolic Medicine and Sciences, The James Black Centre, 125 Coldharbour Lane, London SE5 9NU, UK.

European Heart Journal
|September 15, 2022
PubMed
Summary

Small non-coding RNA therapeutics, including antisense oligonucleotides and miRNA mimics, show promise for cardiovascular diseases. Advances in chemical modifications and delivery methods are improving their efficacy and safety for clinical use.

Keywords:
Antisense oligonucleotidesCardiovascular disordersMicroRNAsRNA therapeuticsShort interfering RNAs

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Cardiovascular Medicine

Background:

  • Small non-coding nucleic acids offer novel therapeutic strategies for various diseases.
  • Cardiovascular diseases represent a significant area of opportunity for RNA-based therapies.
  • Current research focuses on precise, mechanism-based therapeutic approaches.

Approach:

  • Development of antisense oligonucleotides (ASOs) targeting messenger RNAs (mRNAs), microRNAs (miRNAs), or long non-coding RNAs (lncRNAs).
  • Utilizing short interfering RNAs (siRNAs) and miRNA mimics to modulate miRNA pathways.
  • Employing small RNAs, such as aptamers, for protein targeting.

Key Points:

  • Chemical modifications enhance the in vivo properties and reduce immunogenicity of ASOs.
  • Advanced carrier systems (e.g., conjugates, polymers, lipoplexes) improve cellular uptake and nuclease resistance of RNA therapeutics.
  • Several small non-coding RNA therapies for cardiovascular indications are approved, with many more in clinical and preclinical development.

Conclusions:

  • Significant progress has been made in RNA therapeutics for cardiovascular diseases.
  • Ongoing advancements in chemical modifications and delivery systems are crucial for therapeutic success.
  • Overcoming existing hurdles is essential for broader clinical translation of these promising therapies.