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siRNA - Small Interfering RNAs02:30

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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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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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Acute respiratory failure is a condition characterized by the inability of the lungs to perform their primary function: gas exchange. This failure leads to insufficient oxygen levels (hypoxemia) in the blood, elevated carbon dioxide levels (hypercapnia), or both, causing critical impairment in organ function.
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Small Interfering RNA (siRNA) as a Targeted Therapy for Acute Respiratory Distress Syndrome: Evidence from

Viktoriia Kiseleva1,2, Polina Vishnyakova1,2, Andrey Elchaninov1,2,3

  • 1National Medical Research Center for Obstetrics, Gynecology and Perinatology Named after Academician V.I. Kulakov of Ministry of Healthcare of Russian Federation, 117198 Moscow, Russia.

International Journal of Molecular Sciences
|January 28, 2026
PubMed
Summary

Small interfering RNA (siRNA) therapy shows promise for treating Acute Respiratory Distress Syndrome (ARDS). By silencing specific genes, siRNA reduces inflammation and improves lung function, offering a potential pathogenetic treatment for this severe lung condition.

Keywords:
ARDSacute respiratory distress syndromeanimal modelsgene knockdownpreclinical researchsiRNA

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Area of Science:

  • Pulmonary Medicine
  • Molecular Biology
  • RNA Therapeutics

Background:

  • Acute Respiratory Distress Syndrome (ARDS) is a critical lung condition with high mortality.
  • Current treatments for ARDS are limited to supportive care.
  • ARDS pathophysiology involves inflammation, edema, and fibrosis.

Purpose of the Study:

  • To review the potential of small interfering RNA (siRNA) therapy for ARDS.
  • To explore siRNA's mechanism in targeting ARDS-related genes.
  • To summarize preclinical evidence for siRNA in ARDS treatment.

Main Methods:

  • Review of RNA interference mechanisms.
  • Evaluation of animal models for ARDS research (rodents and large animals).
  • Summary of in vivo studies using siRNA for ARDS gene targets.

Main Results:

  • siRNA demonstrates high specificity for gene silencing.
  • Animal models, particularly pigs, offer valuable insights into ARDS.
  • siRNA-mediated gene knockdown (e.g., TIMP1, BTK, TNFα) reduced ARDS inflammation and improved survival in vivo.

Conclusions:

  • siRNA therapy holds significant potential as a pathogenetic treatment for ARDS.
  • Targeting specific genes with siRNA can address the molecular mechanisms of ARDS.
  • This approach may lead to novel therapies beyond supportive care for ARDS.