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相关概念视频

RNA Splicing01:32

RNA Splicing

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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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Alternative RNA Splicing02:18

Alternative RNA Splicing

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Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
There are five types of alternative RNA splicing that vary in the ways the pre-mRNA segments are removed or retained in the mature mRNA. The first...
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Nonsense-mediated mRNA Decay02:27

Nonsense-mediated mRNA Decay

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The Upf proteins that carry out nonsense-mediated decay (NMD) are found in all eukaryotic organisms, including humans. Each protein has an individual role, but they need to work in collaboration. Upf1 is an ATP-dependent RNA helicase that unwinds the RNA helix. Because Upf1 can unwind any RNA, Upf2 and Upf3 are required to help Upf1 discriminate between nonsense and normal mRNAs.
Usually, Upf3 binds to an Exon Junction Complex (EJC) at mRNA splice sites. If a ribosome fully translates the mRNA,...
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What is Gene Expression?01:36

What is Gene Expression?

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A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is comprised  of nucleotides and proteins are comprised of amino acids, a mediator is required to convert the information encoded in DNA into proteins. This mediator is the messenger RNA (mRNA). mRNA copies the blueprint from DNA by a process called transcription. In eukaryotes, transcription occurs in the nucleus by complementary base-pairing with the DNA template. The mRNA is then...
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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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Chromatin Structure Regulates pre-mRNA Processing02:41

Chromatin Structure Regulates pre-mRNA Processing

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In eukaryotic cells, nascent mRNA transcripts need to undergo many post-transcriptional modifications to reach the cell cytoplasm and translate into functional proteins. For a long time, transcription and pre-mRNA processing were considered two independent events that occur sequentially in the cell. However, it has now been well established that transcription and pre-mRNA processing are two simultaneous processes that are precisely regulated inside the cell.
The chromatin structure, especially...
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相关实验视频

Updated: Sep 13, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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预mRNA剪接调节通过反意义的寡核酸.

Natalia N Singh1, Diou Luo2, Ravindra N Singh2

  • 1Department of Biomedical Sciences, College of Veterinary Medicine, Iowa State University, Ames, IA, USA. natalias@iastate.edu.

Methods in molecular biology (Clifton, N.J.)
|July 28, 2025
PubMed
概括
此摘要是机器生成的。

反感性寡核酸 (ASO) 可以调节前传递 RNA (mRNA) 拼接. 这项研究展示了一种基于ASO的策略,用于增强脊髓肌肉缩患者细胞中全长SMN2mRNA的产生.

关键词:
2′-O-甲基修饰的改变无意义的寡核酸 (ASO) 没有意义内部剪接声器N1 (ISS-N1) 是一个内部剪接声器.脊柱肌肉缩症 (SMA) 是指脊柱肌肉缩症.生存运动神经元 (SMN)核作用的核作用.酸胺酸吗啡寡核酸 (PMO) 是一种酸.酸酸的骨干 酸的骨干在mRNA前拼接.转化过程中的转化.

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相关实验视频

Last Updated: Sep 13, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
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A Reporter Based Cellular Assay for Monitoring Splicing Efficiency

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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

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科学领域:

  • 分子生物学分子生物学
  • 遗传学 是一个遗传学.
  • 在RNA生物学,RNA生物学.

背景情况:

  • 预mRNA剪接涉及引子去除和外子结合,由cis元素和RNA结构调节.
  • 拼接 cis 元素是结合特定蛋白质的短图案,影响拼接结果.
  • 反感性寡核酸 (ASO) 可以通过准cis元素或RNA结构来改变拼接.

研究的目的:

  • 开发和评估一种基于ASO的策略,用于治疗拼接的治疗操纵.
  • 在脊髓肌肉缩 (SMA) 患者细胞中增加全长SMN2信使RNA (mRNA) 的产生.

主要方法:

  • 使用的反意义寡核酸 (ASO),旨在与特定的拼接 cis 元素相互作用.
  • 将基于ASO的方法应用于来自脊髓肌肉缩患者的细胞.
  • 量化了全长SMN2mRNA的产生.

主要成果:

  • 描述的基于ASO的方法成功调节了mRNA前剪接.
  • 在SMA患者细胞中显示出全长SMN2mRNA的产生增加.

结论:

  • 基于ASO的策略代表了一个有前途的治疗工具,用于调节像SMA这样的疾病中的拼接.
  • 这种方法提供了一种潜在的方法,通过增加全长的SMN2mRNA来提高功能性SMN蛋白水平.