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相关概念视频

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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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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Drug Discovery: Overview01:26

Drug Discovery: Overview

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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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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相关实验视频

Updated: Jul 11, 2025

MISSION esiRNA for RNAi Screening in Mammalian Cells
15:31

MISSION esiRNA for RNAi Screening in Mammalian Cells

Published on: May 12, 2010

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对于siRNA药物开发的三个"E"挑战

Shuai Guo1, Mengjie Zhang1, Yuanyu Huang2

  • 1School of Life Science, Beijing Institute of Technology, Beijing 100081, China; Advanced Research Institute of Multidisciplinary Science, Beijing Institute of Technology, Beijing 100081, China; Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing 100081, China.

Trends in molecular medicine
|November 11, 2023
PubMed
概括
此摘要是机器生成的。

RNA干扰 (RNAi) 疗法对各种疾病有希望,但面临着交付挑战. 像联体结合剂和组合疗法等策略旨在改善siRNA药物开发和临床转化.

关键词:
药物开发是药物的发展.有效性 有效性 有效性.输入条目 输入条目逃跑 逃跑 逃跑 逃跑 逃跑 逃跑这是一种寡核化物.的siRNA治疗方法.

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Porous Silicon Microparticles for Delivery of siRNA Therapeutics
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Porous Silicon Microparticles for Delivery of siRNA Therapeutics

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Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
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Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

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相关实验视频

Last Updated: Jul 11, 2025

MISSION esiRNA for RNAi Screening in Mammalian Cells
15:31

MISSION esiRNA for RNAi Screening in Mammalian Cells

Published on: May 12, 2010

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Porous Silicon Microparticles for Delivery of siRNA Therapeutics
08:31

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Published on: January 15, 2015

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Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
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科学领域:

  • 生物技术是生物技术.
  • 药理学 药理学是指药理学的学科.
  • 分子生物学分子生物学

背景情况:

  • 小干扰RNA (siRNA) 疗法正在获得引力,有六种已批准的药物和对代谢,心血管,传染病,罕见遗传疾病,癌症和中枢神经系统疾病的广泛调查.
  • 尽管取得了进展,但重大可药性挑战阻碍了基于siRNA的疗法的广泛临床应用.

研究的目的:

  • 讨论siRNA疗法的关键挑战,重点关注"三个E:入口,逃生和有效性.
  • 探索有希望的策略来克服这些障碍,并推动siRNA药物开发向临床转化.

主要方法:

  • 关于siRNA传递和治疗挑战的当前文献的审查和综合.
  • 对新兴策略的分析,包括联体-siRNA合物,新型修饰几何学和组合疗法.

主要成果:

  • 确定了向积累/细胞吸收 ("入口"),内解体逃脱 ("逃脱") 和体内表现 ("有效性") 作为关键障碍.
  • 突出了多样化的联结体-siRNA联结体,扩大了疾病点,新的修饰几何学和组合疗法作为有前途的解决方案.

结论:

  • 解决"三E"挑战对于成功的siRNA治疗开发至关重要.
  • 拟议的战略为改善临床转化和扩大siRNA药物的应用提供了潜在的途径.