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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.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
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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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Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
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Related Experiment Video

Updated: May 12, 2025

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
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Recent Update on siRNA Therapeutics.

Oluwakemi Ebenezer1, Abel Kolawole Oyebamiji2, John Oludele Olanlokun3

  • 1Department of Physics, University of Alberta, Edmonton, AB T6G 2E1, Canada.

International Journal of Molecular Sciences
|May 7, 2025
PubMed
Summary
This summary is machine-generated.

Small interfering RNA (siRNA) offers a powerful approach to treat diseases by silencing specific genes. This review details siRNA technology, delivery challenges, and clinical progress in areas like cancer and genetic disorders.

Keywords:
clinical trialdiseasesgene therapysiRNAsiRNA therapeuticstri-GalNAc conjugates

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Therapeutics

Background:

  • Small interfering RNA (siRNA) represents a novel therapeutic modality targeting gene silencing.
  • siRNA therapeutics operate post-transcriptionally, selectively inhibiting disease-causing genes.

Purpose of the Study:

  • To provide a comprehensive overview of current siRNA-based therapeutics.
  • To highlight the potential applications of siRNA in various disease areas.
  • To discuss challenges and strategies in siRNA development and clinical translation.

Main Methods:

  • Review of siRNA technology principles, including mechanism of action and delivery systems.
  • Analysis of impediments in siRNA delivery and manufacturing.
  • Summary of clinical advancements across diverse therapeutic fields.

Main Results:

  • siRNA technology demonstrates significant potential in treating cancer, genetic disorders, viral infections, and inflammatory diseases.
  • Key challenges in siRNA delivery and manufacturing have been identified.
  • Strategies to overcome these obstacles are being developed and implemented.

Conclusions:

  • Advancements in siRNA delivery systems and target selection are crucial for revolutionizing medicine.
  • siRNA therapeutics hold promise for offering new treatment options for patients with various conditions.
  • The field is progressing, with lessons learned from successes and failures guiding future development.