Jove
Visualize
联系我们
JoVE
x logofacebook logolinkedin logoyoutube logo
关于 JoVE
概览领导团队博客JoVE 帮助中心
作者
出版流程编辑委员会范围与政策同行评审常见问题投稿
图书馆员
用户评价订阅访问资源图书馆顾问委员会常见问题
研究
JoVE JournalMethods CollectionsJoVE Encyclopedia of Experiments存档
教育
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab Manual教师资源中心教师网站
使用条款与条件
隐私政策
政策

相关概念视频

RNA Editing02:23

RNA Editing

8.9K
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...
8.9K
Urea Cycle01:23

Urea Cycle

43.9K
The urea cycle describes how liver cells convert ammonia to urea. Ammonia is a toxic waste product of protein catabolism. Land animals must convert ammonia into the less toxic urea which can be safely eliminated by the kidneys through urine. Marine animals excrete ammonia directly, and the surrounding water dilutes the ammonia to safe levels.
43.9K
Experimental RNAi02:15

Experimental RNAi

6.1K
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...
6.1K
Types of RNA01:23

Types of RNA

63.3K
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...
63.3K
RNA Stability01:53

RNA Stability

33.3K
Intact DNA strands can be found in fossils, while scientists sometimes struggle to keep RNA intact under laboratory conditions. The structural variations between RNA and DNA underlie the differences in their stability and longevity. Because DNA is double-stranded, it is inherently more stable. The single-stranded structure of RNA is less stable but also more flexible and can form weak internal bonds. Additionally, most RNAs in the cell are relatively short, while DNA can be up to 250 million...
33.3K
Translation01:31

Translation

14.6K
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...
14.6K

您也可能阅读

相关文章

通过共同作者、期刊和引用图与本文相关的文章。

排序
Same author

Oligonucleotides targeting the 3' splice site downstream of a microexon as an innovative therapy for autism.

NAR molecular medicine·2025
Same author

Propionic Acidemia-Induced Proarrhythmic Electrophysiological Alterations in Human iPSC-Derived Cardiomyocytes.

Journal of inherited metabolic disease·2025
Same author

Novel CRISPR-Cas9 iPSC knockouts for PCCA and PCCB genes: advancing propionic acidemia research.

Human cell·2025
Same author

Renal phenotyping in a hypomorphic murine model of propionic aciduria reveals common pathomechanisms in organic acidurias.

Scientific reports·2024
Same author

Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria.

Nucleic acid therapeutics·2024
Same author

PAH deficient pathology in humanized c.1066-11G>A phenylketonuria mice.

Human molecular genetics·2024

相关实验视频

Updated: Jun 9, 2025

RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes
09:19

RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes

Published on: July 22, 2014

8.4K

探索用于尿素循环障碍的RNA疗法.

Eva Richard1, Ainhoa Martínez-Pizarro1, Lourdes R Desviat1

  • 1Centro de Biología Molecular Severo Ochoa UAM-CSIC, IUBM, CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.

Journal of inherited metabolic disease
|October 25, 2024
PubMed
概括

基于RNA的疗法为尿素循环障碍 (UCD) 提供了新的希望,这是一组严重的肝脏疾病. 脂质纳米颗粒配制的信使RNA (mRNA) 疗法在几种UCD的临床前研究中显示出前景,其中一种正在进入临床试验.

科学领域:

  • 生物化学 生物化学
  • 遗传学 遗传学 是一个
  • 药理学 药理学是指药理学的学科.

背景情况:

  • RNA疗法代表了医学上的范式转变,为基因表达调制提供了新的策略.
  • 使者RNA (mRNA) 疫苗的成功加速了RNA研究和开发.
  • 尿素循环障碍 (UCD) 是一种严重的遗传性肝病,治疗选择有限.

研究的目的:

  • 审查基于RNA的药物治疗尿素循环障碍 (UCD) 的潜力.
  • 探索针对特定尿素循环缺陷的RNA应用的持续发展.
  • 突出肝脏基因向传递系统的进步.

主要方法:

  • 对UCDs的基于RNA的疗法进行的临床前和临床研究的审查.
  • 分析各种RNA模式,包括mRNA,siRNA等.
  • 输送系统的评估,如脂质纳米颗粒 (LNP) 和N-乙黄胺 (GalNAc) 结合物.

主要成果:

  • 一些基于RNA的治疗策略正在对UCD进行研究.
  • 脂质纳米颗粒 (LNP) 配方的mRNA疗法已在多种UCD中显示出临床前的疗效,包括氨酸缺乏和氨酸酶缺乏.
  • 甲尼丁转糖胺酶缺乏症是第一个进入临床试验的LNP配方mRNA疗法的UCD.
关键词:
反感的寡核化物.mRNA疗法是一种mRNA疗法.甲酸转碳胺酶缺乏症 甲酸转碳胺酶缺乏症这是一个假的exonon.拼接 拼接 拼接 拼接尿素循环中的尿素.

更多相关视频

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
12:03

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

7.8K
Investigation of RNA Synthesis Using 5-Bromouridine Labelling and Immunoprecipitation
09:59

Investigation of RNA Synthesis Using 5-Bromouridine Labelling and Immunoprecipitation

Published on: May 3, 2018

9.5K

相关实验视频

Last Updated: Jun 9, 2025

RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes
09:19

RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes

Published on: July 22, 2014

8.4K
Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
12:03

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

7.8K
Investigation of RNA Synthesis Using 5-Bromouridine Labelling and Immunoprecipitation
09:59

Investigation of RNA Synthesis Using 5-Bromouridine Labelling and Immunoprecipitation

Published on: May 3, 2018

9.5K

结论:

  • 基于RNA的药物,特别是LNP配方的mRNA,对于尿素循环障碍具有显著的治疗潜力.
  • 针对性地将遗传有效载荷传递到肝脏对于UCD治疗至关重要.
  • RNA技术的进步为罕见遗传性肝病的创新治疗铺平了道路.