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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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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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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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RNA-seq03:21

RNA-seq

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RNA sequencing, or RNA-Seq, is a high-throughput sequencing technology used to study the transcriptome of a cell. Transcriptomics helps to interpret the functional elements of a genome and identify the molecular constituents of an organism. Additionally, it also helps in understanding the development of an organism and the occurrence of diseases. 
Before the discovery of RNA-seq, microarray-based methods and Sanger sequencing were used for transcriptome analysis. However, while...
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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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Ribosome Profiling02:24

Ribosome Profiling

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Ribosome profiling or ribo-sequencing is a deep sequencing technique that produces a snapshot of active translation in a cell. It selectively sequences the mRNAs protected by ribosomes to get an insight into a cell’s translation landscape at any given point in time.
Applications of ribosome profiling
Ribosome profiling has many applications, including in vivo monitoring of translation inside a particular organ or tissue type and quantifying new protein synthesis levels.
The technique...
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RNAi-Based Therapeutics and Novel RNA Bioengineering Technologies.

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RNA interference (RNAi) offers gene modulation via microRNAs (miRNAs) and small interfering RNAs (siRNAs). This review examines RNAi therapeutics, approved drugs, and novel biologic agents for research and development.

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • RNA interference (RNAi) is a versatile tool for modulating gene expression using microRNAs (miRNAs) and small interfering RNAs (siRNAs).
  • RNAi therapeutics have advanced significantly, with several drugs approved and more in clinical trials, demonstrating potential for treating various diseases.
  • Chemical modifications, while enhancing stability and efficacy, can introduce off-target effects, necessitating exploration of alternative approaches.

Purpose of the Study:

  • To review the mechanisms of action for endogenous miRNAs and exogenous siRNAs.
  • To examine the challenges and advancements in RNAi drug development, including delivery barriers and chemical modifications.
  • To overview the pharmacology of approved RNAi medications and those in clinical trials, highlighting novel biologic RNAi agents.

Main Methods:

  • Literature review of RNA interference mechanisms, therapeutic strategies, and drug development.
  • Analysis of FDA-approved siRNA medications and ongoing clinical trials for siRNA and miRNA-based therapeutics.
  • Discussion of novel technologies for producing biologic RNAi agents using intact cells.

Main Results:

  • Four siRNA medications are FDA-approved, with numerous other RNAi therapeutics progressing through clinical trials.
  • Chemical modifications in RNAi agents can impact stability and efficacy but may also increase off-target effects.
  • Emerging technologies focus on creating true biologic RNAi agents with improved safety and efficacy profiles.

Conclusions:

  • RNAi technology is a powerful therapeutic strategy with ongoing advancements in drug development.
  • Addressing delivery barriers and minimizing off-target effects are crucial for successful RNAi therapeutics.
  • Novel approaches for producing biologic RNAi agents hold promise for future research and clinical applications.