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相关概念视频

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-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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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.
The aptamer has high specificity for a particular metabolite which allows riboswitches to specifically regulate...
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Leaky Scanning02:28

Leaky Scanning

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During most eukaryotic translation processes, the small 40S ribosome subunit scans an mRNA from its 5' end until it encounters the first start AUG codon. The large 60S ribosomal subunit then joins the smaller one to initiate protein synthesis. The location of the translation initiation is largely determined by the nucleotides near the start codon as there may be multiple translation initiation sites present on the mRNA.  Marilyn Kozak discovered that the sequence RCCAUGG (where R...
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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

16.7K
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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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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相关实验视频

Updated: Jun 26, 2025

Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations
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Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations

Published on: August 4, 2016

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对于RNA疗法的发现平台.

Giulio Ciucci1, Luca Braga2, Serena Zacchigna1,3

  • 1Cardiovascular Biology Laboratory, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.

British journal of pharmacology
|May 18, 2024
PubMed
概括

本综述详细介绍了用于发现RNA治疗药物的技术平台,重点关注到达高级临床阶段或市场批准的技术平台. 它强调了针对以前无法治疗的疾病的方法.

关键词:
对于RNA疗法来说,它是非常重要的.发现药物的发现.功能查功能查

更多相关视频

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 Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins
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Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins

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相关实验视频

Last Updated: Jun 26, 2025

Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations
11:52

Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations

Published on: August 4, 2016

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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 Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins
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Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins

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科学领域:

  • 生物技术和制药科学 生物技术和制药科学
  • 分子生物学和遗传学分子生物学和遗传学

背景情况:

  • RNA疗法为以前被认为是无法治疗的疾病提供了新的策略.
  • 现有的审查广泛涵盖RNA治疗优势和交付,但缺乏关注发现平台.

研究的目的:

  • 为RNA治疗发现提供技术平台和ex vivo试验的全面概述.
  • 分析各种RNA发现方法的优缺点.
  • 要突出RNA疗法已经达到先进的临床试验阶段或市场批准.

主要方法:

  • 对现有的RNA发现平台和ex vivo测试的文献综述.
  • 分析平台在RNA治疗开发中的适用性.
  • 专注于RNA疗法在第三阶段试验或经过市场批准.

主要成果:

  • 识别和分类RNA发现的各种技术平台.
  • 评估了不同的ex vivo试验的优点和局限性.
  • 提供了关于这些平台在成功的RNA疗法中的应用的见解.

结论:

  • 技术平台对于推动RNA疗法从发现到临床应用至关重要.
  • 了解这些平台是开发挑战性疾病新疗法的关键.