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Related Concept Videos

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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Types of RNA01:20

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 regulating 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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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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RNA Interference01:23

RNA Interference

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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.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
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Translational Regulation01:29

Translational Regulation

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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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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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Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells
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Noncoding RNA therapeutics - challenges and potential solutions.

Melanie Winkle1, Sherien M El-Daly2, Muller Fabbri3

  • 1Translational Molecular Pathology, MD Anderson Cancer Center, Texas State University, Houston, TX, USA.

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RNA therapeutics offer promising treatments for diseases like cancer. Current challenges in specificity, delivery, and tolerability are being addressed by emerging approaches to improve efficacy and patient outcomes.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Pharmacology

Background:

  • Noncoding RNAs (ncRNAs), including microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), are crucial in disease pathogenesis.
  • RNA-based therapeutics, primarily antisense oligonucleotides and small interfering RNAs, have shown potential but face challenges in clinical application.
  • Existing RNA therapeutics have yielded mixed results regarding efficacy and safety, necessitating further development.

Purpose of the Study:

  • To review the current landscape of noncoding RNA therapeutics.
  • To identify key challenges hindering the clinical success of ncRNA-based drugs.
  • To highlight novel strategies aimed at enhancing the effectiveness and safety of ncRNA therapies.

Main Methods:

  • Literature review of current research and clinical trials on ncRNA therapeutics.
  • Analysis of challenges including specificity, delivery methods, and patient tolerability.
  • Exploration of emerging therapeutic modalities and approaches.

Main Results:

  • Several RNA-based therapeutics have gained regulatory approval, but clinical outcomes remain variable.
  • Specificity, efficient delivery to target tissues, and managing potential toxicity are significant hurdles.
  • Promising new strategies are emerging to overcome these limitations and improve therapeutic potential.

Conclusions:

  • Noncoding RNA therapeutics hold significant promise for treating various diseases, including cancer.
  • Addressing challenges in specificity, delivery, and tolerability is critical for advancing ncRNA-based treatments.
  • Emerging approaches offer new avenues to enhance the success of ncRNA therapeutics in clinical practice.