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相关概念视频

RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Pre-mRNA Processing: Modification of pre-mRNA Ends01:35

Pre-mRNA Processing: Modification of pre-mRNA Ends

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In eukaryotic cells, transcripts made by RNA polymerase are modified and processed before exiting the nucleus. Unprocessed RNA is called precursor mRNA or pre-mRNA to distinguish it from mature mRNA.
Once about 20-40 ribonucleotides have been joined together by RNA polymerase, a group of enzymes adds a cap to the 5' end of the growing transcript. In this process, a 5' phosphate is replaced by modified guanosine that has a methyl group attached (7-methyl guanosine). This 5' cap helps...
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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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相关实验视频

Updated: Jun 23, 2025

A Nonsequencing Approach for the Rapid Detection of RNA Editing
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使用深度学习优化5'UTR用于mRNA传递的基因编辑.

Sebastian Castillo-Hair1,2, Stephen Fedak3, Ban Wang4

  • 1Department of Electrical & Computer Engineering, University of Washington, Seattle, WA, USA.

Nature communications
|June 20, 2024
PubMed
概括
此摘要是机器生成的。

深度学习模型优化信使RNA (mRNA) 5'未翻译区域 (UTR) 以增强蛋白质表达. 设计的UTR增强了基因编辑酶活性,显示了mRNA治疗的前景.

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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
  • 生物信息学是一种生物信息学.

背景情况:

  • 使者RNA (mRNA) 疗法提供了革命性的潜力,但缺乏用于增加表达的序列优化方法.
  • 有效的mRNA翻译对于治疗疗效至关重要,但序列设计仍然是一个挑战.

研究的目的:

  • 使用深度学习设计5'未翻译区域 (UTR) 以实现高效的mRNA翻译.
  • 评估设计的5'UTRs在支持基因编辑酶表达和活性方面的性能.

主要方法:

  • 利用深度学习,梯度下降和生成神经网络进行5'UTR序列设计.
  • 在三种细胞类型的随机5'UTR图书馆上进行了多元体分析,以生成训练数据.
  • 经过实验测试,设计的5'UTR与mRNA编码大TAL基因编辑酶在两个细胞系和两个基因标中进行了实验.

主要成果:

  • 5'UTR的表现显示了不同细胞类型的高相关性.
  • 设计的5'UTR显著增强了基因编辑活动.
  • 虽然编辑效率显示了跨细胞类型和跨目标相关性,但最佳的UTR性能是特定于环境的.

结论:

  • 基于模型的序列设计具有优化mRNA治疗的巨大潜力.
  • 深度学习方法可以有效地指导高性能mRNA序列的设计.
  • 开发的方法有助于创建更强效和高效的基于mRNA的疗法.