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Related Concept Videos

RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Urea Cycle01:23

Urea Cycle

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The urea cycle describes how liver cells convert ammonia to urea. Ammonia is a toxic waste product of protein catabolism. Land animals must convert ammonia into the less toxic urea which can be safely eliminated by the kidneys through urine. Marine animals excrete ammonia directly, and the surrounding water dilutes the ammonia to safe levels.
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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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Types of RNA01:23

Types of RNA

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Overview
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.
RNA...
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RNA Stability01:53

RNA Stability

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Intact DNA strands can be found in fossils, while scientists sometimes struggle to keep RNA intact under laboratory conditions. The structural variations between RNA and DNA underlie the differences in their stability and longevity. Because DNA is double-stranded, it is inherently more stable. The single-stranded structure of RNA is less stable but also more flexible and can form weak internal bonds. Additionally, most RNAs in the cell are relatively short, while DNA can be up to 250 million...
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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.
Translation Produces the Building Blocks of Life
Proteins are...
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Related Experiment Video

Updated: Jun 9, 2025

RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes
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RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes

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Exploring RNA therapeutics for urea cycle disorders.

Eva Richard1, Ainhoa Martínez-Pizarro1, Lourdes R Desviat1

  • 1Centro de Biología Molecular Severo Ochoa UAM-CSIC, IUBM, CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.

Journal of Inherited Metabolic Disease
|October 25, 2024
PubMed
Summary
This summary is machine-generated.

RNA-based therapies offer new hope for urea cycle disorders (UCD), a group of serious liver diseases. Lipid nanoparticle-formulated messenger RNA (mRNA) therapies are showing promise in preclinical studies for several UCDs, with one advancing to clinical trials.

Keywords:
antisense oligonucleotidesmRNA therapyornithine transcarbamylase deficiencypseudoexonsplicingurea cycle

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Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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Area of Science:

  • Biochemistry
  • Genetics
  • Pharmacology

Background:

  • RNA therapeutics represent a paradigm shift in medicine, offering novel strategies for gene expression modulation.
  • The success of messenger RNA (mRNA) vaccines has accelerated RNA research and development.
  • Urea cycle disorders (UCDs) are severe genetic liver diseases with limited treatment options.

Purpose of the Study:

  • To review the potential of RNA-based medicines for treating urea cycle disorders (UCDs).
  • To explore ongoing developments in RNA applications targeting specific urea cycle defects.
  • To highlight advancements in delivery systems for hepatic gene targeting.

Main Methods:

  • Review of preclinical and clinical studies on RNA-based therapies for UCDs.
  • Analysis of various RNA modalities including mRNA, siRNA, and others.
  • Evaluation of delivery systems such as lipid nanoparticles (LNPs) and N-acetylgalactosamine (GalNAc) conjugates.

Main Results:

  • Several RNA-based therapeutic strategies are under investigation for UCDs.
  • Lipid nanoparticle (LNP)-formulated mRNA therapy has shown preclinical efficacy for multiple UCDs, including citrin deficiency and arginase deficiency.
  • Ornithine transcarbamylase deficiency is the first UCD to enter clinical trials with LNP-formulated mRNA therapy.

Conclusions:

  • RNA-based medicines, particularly LNP-formulated mRNA, hold significant therapeutic potential for urea cycle disorders.
  • Targeted delivery of genetic payloads to the liver is crucial for UCD treatment.
  • Advancements in RNA technology pave the way for innovative treatments for rare genetic liver diseases.