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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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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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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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Types of RNA01:23

Types of RNA

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Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
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Translation01:31

Translation

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Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
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Related Experiment Video

Updated: Aug 13, 2025

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
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What Can RNA-Based Therapy Do for Monogenic Diseases?

Luka A Clarke1, Margarida D Amaral1

  • 1BioISI-Biosystems & Integrative Sciences Institute, Faculty of Sciences, University of Lisboa, 1749-016 Lisboa, Portugal.

Pharmaceutics
|January 21, 2023
PubMed
Summary

RNA-based therapies offer new treatments for monogenic diseases by modulating RNA processing with antisense oligonucleotides (ASOs) or delivering messenger RNA (mRNA). These promising strategies require further optimization for delivery, safety, and efficacy.

Keywords:
antisense oligonucleotidesmRNAmonogenic disorderspersonalized medicinepremature termination codon mutationssplicing mutations

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Area of Science:

  • Biotechnology
  • Genetics
  • Molecular Biology

Background:

  • Monogenic diseases are hereditary disorders caused by mutations in single genes.
  • RNA-based therapeutic approaches are emerging as potential treatments for these conditions.
  • Current strategies include antisense oligonucleotides (ASOs) and messenger RNA (mRNA) administration.

Purpose of the Study:

  • To review and summarize the current RNA-based therapeutic strategies for monogenic diseases.
  • To highlight the mechanisms of action for ASOs and mRNA therapies.
  • To identify areas for future optimization in RNA-based treatments.

Main Methods:

  • Antisense oligonucleotides (ASOs) modulate RNA processing, including splicing and exon skipping.
  • ASOs can prevent degradation of messenger RNA (mRNA) or induce degradation of toxic mRNA.
  • Messenger RNA (mRNA) therapy, similar to gene therapy, is mutation-agnostic and targets the cytoplasm.

Main Results:

  • ASO approaches offer versatile RNA processing modulation for various genetic disorders.
  • mRNA therapy presents a simpler, mutation-agnostic approach for recessive monogenic diseases.
  • Both strategies show significant promise, evidenced by applications like COVID-19 vaccines.

Conclusions:

  • RNA-based therapies, including ASOs and mRNA, represent a significant advancement in treating monogenic diseases.
  • Further research is needed to enhance delivery efficiency and reduce potential adverse effects and toxicity.
  • Optimization of these RNA-based strategies holds great potential for broader clinical application.