开发具有先进准功能的mRNA纳米药物.
Ji Wang1,2, Lijun Cai2, Ning Li2
1Department of Radiology, Nanjing Drum Tower Hospital, Medical School, Nanjing University, Nanjing, 210008, People's Republic of China.
Nano-micro letters
|February 20, 2025
概括
向信使RNA (mRNA) 纳米药物通过将mRNA传递到特定的部位来提供精确的疾病治疗. 本综述探讨了这些先进疗法的向策略,生物障碍和临床转化.
科学领域:
- 生物医学工程 生物医学工程
- 纳米技术 纳米技术
- 分子医学是分子医学.
背景情况:
- 使者RNA (mRNA) 纳米药物正在成为疾病治疗的强大平台.
- 针对性地提供mRNA纳米药物提高了治疗效率,并最大限度地减少了目标外的副作用.
- 最近的进展侧重于开发精确的器官和组织向的复杂策略.
研究的目的:
- 提供对具有向功能的mRNA纳米药物最新研究的全面审查.
- 阐明被动性,内源性和活性向mRNA输送的机制.
- 讨论生物障碍,器官向策略和mRNA纳米药物的临床翻译挑战.
主要方法:
- 详细描述mRNA及其各种载体.
- 被动性,内源性和主动性准机制的概述.
- 在体内分娩过程中遇到的生物障碍的分析.
- 基于mRNA的疗法的器官向策略的总结.
主要成果:
- 确定关键的向机制及其相关的生物障碍.
- 对用于器官特异性mRNA输送的各种策略的概述.
- 在临床环境中讨论mRNA纳米药物的现状和未来前景.
结论:
- 向mRNA纳米药物代表了精准医学的重大进步.
- 克服生物障碍和优化准策略对于临床翻译至关重要.
- 对mRNA向技术的进一步研究为未来的治疗应用提供了巨大的潜力.
相关概念视频
MicroRNAs
3.0K
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
3.0K
Experimental RNAi
6.0K
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...
6.0K
siRNA - Small Interfering RNAs
16.5K
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...
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...
16.5K
Types of RNA
63.0K
Overview
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...
63.0K
Regulated mRNA Transport
6.2K
In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing...
6.2K
Regulation of Expression Occurs at Multiple Steps
22.4K
Gene expression can be regulated at almost every step from gene to protein. Transcription is the step that is most commonly regulated. This involves the binding of proteins to short regulatory sequences on the DNA. This association can either promote or inhibit the transcription of a gene associated with the respective sequence.
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
22.4K


