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相关概念视频

Experimental RNAi02:15

Experimental RNAi

6.0K
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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Regulated mRNA Transport02:22

Regulated mRNA Transport

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In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing...
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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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Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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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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选择性表达的RNA分子作为功能化的细胞向的多功能工具.

Frederik Rastfeld1, Marco Hoffmann1, Sylvie Krüger1

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概括

研究人员开发了新的RNA分子,用于精确的细胞向和治疗. 这些选择性表达的RNA分子 (seRNAs) 仅在细胞中激活,为治疗乳腺癌和质母细胞瘤等疾病提供了一个新的平台.

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科学领域:

  • 生物技术是生物技术.
  • 分子生物学分子生物学
  • 在RNA治疗方面,RNA疗法.

背景情况:

  • 精确准患病细胞对于下一代药品至关重要.
  • 目前的方法往往由于缺少细胞上特定的表面标记物而失败.

研究的目的:

  • 开发一种基于RNA的新方法,用于精确的细胞向和选择性效应蛋白表达.
  • 为治疗和诊断应用创建一个多功能平台技术.

主要方法:

  • 通过结合反意义技术,mRNA疗法和功能性RNA二次结构来开发选择性表达的RNA分子 (seRNAs).
  • 设计seRNA在非目标细胞中保持不活跃,并在预先选择的细胞类型中通过部分降解来激活.
  • 使用模块化系统,以实现可适应的细胞特异性和功能化.

主要成果:

  • 证明了seRNAs作为高度选择性的细胞向平台的概念验证.
  • 在混合细胞系统中成功治疗乳腺瘤细胞群.
  • 在雄性小鼠的大脑中有效减少U87质母细胞瘤细胞群,没有可观察到的副作用.

结论:

  • 开发的seRNA技术可以实现精确的细胞向和选择性治疗蛋白质表达.
  • 该平台具有广泛的治疗应用的巨大潜力,包括癌症治疗.
  • 模块化设计允许轻松适应各种细胞类型和医疗需求.