用小分子准RNA,从RNA结构到精密药物:IUPHAR评论:40
Yuquan Tong1,2, Jessica L Childs-Disney2, Matthew D Disney1,2
1Department of Chemistry, The Scripps Research Institute, Jupiter, Florida, USA.
British journal of pharmacology
|September 3, 2024
概括
针对结构化RNA的小分子为疾病提供治疗潜力. 本综述涵盖了开发这些RNA向药物的策略,并解决了它们临床转化方面的挑战.
科学领域:
- 分子生物学分子生物学
- 药用化学 医学化学
- 药物发现 药物发现 药物发现
背景情况:
- 核糖核酸 (RNA) 结构对于健康和疾病中的生物功能至关重要.
- 用小分子向结构化RNA是一种有前途的治疗策略.
- 在RNA生物学和结构确定方面的进步加速了RNA向小分子的发展.
研究的目的:
- 审查研究RNA结构和识别与它们结合的小分子的策略.
- 要总结最近在开发RNA向小分子作为治疗药物的进展.
- 确定挑战并提出RNA结合小分子临床翻译的路线图.
主要方法:
- 关于RNA结构确定和小分子识别的文献综述.
- 对RNA向药物设计和治疗应用的最新发展进行分析.
- 讨论该领域的挑战和未来方向.
主要成果:
- 针对结构化RNA的小分子可以作为化学探针和治疗线索.
- 存在各种策略来识别和设计RNA结合小分子.
- 在开发针对不同疾病的RNA向疗法方面取得了重大进展.
结论:
- 向RNA的小分子具有很大的治疗前景,但面临临临床开发的障碍.
- 克服药物输送,特异性和临床验证方面的挑战是必不可少的.
- 未来的研究应该集中在弥合发现和基于RNA的药物的临床应用之间的差距.
关键词:
这是一个RNARNARNARNARNA.喜梅拉斯 (Chimeras) 是一种幻象.药物设计 药物设计它们是异性双功能的.诱导近距离的诱导近距离小分子的小分子.目标降解降解的目标降解.转录组 (transcriptome) 是一个转录组.更多相关视频
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Types of RNA
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Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...
RNA Interference
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...
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...
RNA Interference
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...
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...
siRNA - Small Interfering RNAs
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 ATP-dependent...
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 ATP-dependent...
Experimental RNAi
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...
Types of RNA
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in regulating gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA Performs Diverse...
RNA Performs Diverse...
