相关实验视频

Updated: May 27, 2025

In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells

In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells

Published on: November 13, 2014

使用聚adenosine尾部模仿剂来增强与哈普隆缺陷障碍相关的基因的mRNA表达

Bahareh Torkzaban¹, Yining Zhu^2,3, Christian Lopez¹

¹Department of Molecular Biology and Genetics, Johns Hopkins University, Baltimore, MD 21205, USA.

Molecular therapy. Nucleic acids

|February 19, 2025

概括

研究人员开发了mRNA增强剂,通过模仿多甲尾来增强基因表达. 这项技术通过增加向基因的蛋白质生产来治疗哈普洛因缺乏症疾病的前景.

科学领域:

分子生物学分子生物学
遗传学遗传学是一个
生物技术是生物技术.

背景情况:

使者RNA (mRNA) 上的多氨酸 (poly(A)) 尾巴对于稳定性和翻译至关重要.
在细胞质中控制地去除多A尾,调节基因表达,并可能导致mRNA降解.
哈普洛缺乏症疾病是由减少基因表达引起的,影响细胞功能.

研究的目的:

开发一种新的治疗策略,以增强mRNA的表达.
调查聚甲尾模仿剂在治疗遗传疾病方面的潜力.
为了证明mRNA增强剂在增强基因表达中的有效性.

主要方法:

设计和合成短RNA序列与多种类型的尾巴模拟器.
反感杂交到目标mRNAs的3'未翻译区域 (UTR).
在细胞培养和动物模型中增强mRNA表达的体外和体内验证.

主要成果:

选择性和显著的目标mRNA表达的增强是通过使用多种类型的尾巴模仿物来实现的.
这项被称为mRNA增强剂的技术有效地增加了与自闭症谱系障碍相关的基因表达 (例如,SYNGAP1,MECP2,PURA,CTNNB1).
在人类细胞培养和动物模型中观察到基因表达的增加.

结论:

小型多甲尾模仿剂可以显著增强mRNA表达.
mRNA增强剂代表了对哈普隆不足障碍的有前途的治疗方式.
这种方法提供了一个潜在的策略来治疗基因条件,其特点是基因剂量不足.

关键词:

MT:寡核酸:治疗方法和应用.对于RNA疗法来说,它是一种治疗方法.反感的寡核化物.基因表达的基因表达方式哈普洛因缺乏障碍哈普洛因缺乏障碍 mRNA的表达方式聚亚脱化聚亚脱化转录后监管的规定.

更多相关视频

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation

Published on: February 1, 2019

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

相关概念视频

Experimental RNAi

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...

mRNA Stability and Gene Expression

mRNA Stability and Gene Expression

The structure and stability of mRNA molecules regulates gene expression, as mRNAs are a key step in the pathway from gene to protein. In eukaryotes, the half-life of mRNA varies from a few minutes up to several days. mRNA stability is essential in growth and development. The absence of the proteins regulating its stability, such as tristetraprolin in mice, can cause systemic issues, including bone marrow overgrowth, inflammation, and autoimmunity.
Cis-acting Elements involved in mRNA stability

RNA Editing

RNA Editing

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...

Nonsense-mediated mRNA Decay

Nonsense-mediated mRNA Decay

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,...

Translation

Translation

Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
Translation Produces the Building Blocks of Life
Proteins are...

Alternative RNA Splicing

Alternative RNA Splicing

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...