相关实验视频
Updated: Jun 27, 2025

08:53
A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
Published on: September 15, 2021
2.7K
调节RNA拼接的小分子:对目标和未来前景的审查
Léa Bouton1,2, Agathe Ecoutin1,2, Florian Malard1,2
1Inserm U1212, CNRS UMR5320, ARNA Laboratory, University of Bordeaux 146 rue Léo Saignat 33076 Bordeaux Cedex France florian.malard@inserm.fr sebastien.campagne@inserm.fr.
RSC medicinal chemistry
|April 26, 2024
概括
小有机分子提供了一种有希望的新方法来纠正异常的RNA剪接,这是基因表达中的一个关键过程,与癌症等疾病有关. 这些分子克服了较旧的反感性寡核酸的局限性,增强了治疗潜力.
科学领域:
- 分子生物学分子生物学
- 遗传学 是一个遗传学.
- 药物发现 药物发现 药物发现
背景情况:
- RNA剪接对于真核生物的基因表达至关重要;它的失调会导致疾病,包括遗传性疾病和癌症.
- 传统的反感性寡核酸可纠正拼接错误,但具有诸如口服生物可用性和稳定性差等局限性.
- 开发小型有机分子为调节RNA拼接提供了一个替代策略.
研究的目的:
- 审查针对小分子的当前和潜在的治疗点,旨在纠正异常RNA拼接.
- 讨论RNA向小分子在拼接校正中的治疗应用.
- 为RNA剪接调节的策略提供最新信息.
主要方法:
- 关于RNA拼接调制的最新进展的文献综述.
- 讨论小分子干预的已知和潜在的分子点.
- 分析涉及小有机分子的治疗策略.
主要成果:
- 小的有机分子正在出现作为一种可行的替代品,用于拼接校正的反意义寡核酸.
- 确定的目标包括RNA结构,跨作用拼接因子和拼接体.
- 最近的进展强调了小分子在纠正拼接缺陷方面的治疗前景.
结论:
- 小有机分子在纠正异常RNA拼接方面显示出显著的治疗潜力.
- 用小分子准RNA结构,拼接因子和拼接体是一个有前途的治疗途径.
- 这篇评论强调了RNA拼接调制的不断变化的格局和小分子治疗的未来.
相关概念视频
RNA Splicing
56.3K
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...
56.3K
Alternative RNA Splicing
21.1K
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...
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...
21.1K
Chromatin Structure Regulates pre-mRNA Processing
7.0K
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...
The chromatin structure, especially...
7.0K
Chromatin Structure and RNA Splicing
2.7K
2.7K
What is Gene Expression?
8.5K
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...
8.5K
Regulation of Expression at Multiple Steps
899
The gene expression in cells is regulated at different stages: (i) transcription, (ii) RNA processing, (iii) RNA localization, and (iv) translation. Transcriptional regulation is mediated by regulatory proteins such as transcription factors, activators, or repressors—these control gene expression by initiating or inhibiting the transcription of genes. Once a precursor or pre-mRNA is produced, it undergoes post-transcriptional modification, including 5' capping, splicing, and the...
899

