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相关概念视频

RNA Splicing01:32

RNA Splicing

60.6K
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...
60.6K
Alternative RNA Splicing02:18

Alternative RNA Splicing

25.1K
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...
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Alternative RNA Splicing02:18

Alternative RNA Splicing

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No description available
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Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
27.6K
Chromatin Structure and RNA Splicing02:41

Chromatin Structure and RNA Splicing

3.4K
No description available
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Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy01:30

Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy

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Various diagnostic tests are employed in the diagnostic process for Inflammatory Bowel Disease (IBD), particularly to differentiate between Crohn's disease and ulcerative colitis.
Diagnostic studies
A colonoscopy is the definitive screening test, distinguishing ulcerative colitis from other colon diseases with similar symptoms. During a colonoscopy test, inflamed mucosa with exudate ulcerations can be observed, and biopsies are taken to determine the histologic characteristics of the...
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相关实验视频

Updated: Jan 31, 2026

Evaluation of Cardiac Contractility Modulation Therapy in 2D Human Stem Cell-Derived Cardiomyocytes
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Evaluation of Cardiac Contractility Modulation Therapy in 2D Human Stem Cell-Derived Cardiomyocytes

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对于人类疾病的拼接调节疗法.

Pietro Spitali1, Annemieke Aartsma-Rus

  • 1Department of Human Genetics, Leiden University Medical Center, 2300 RC Leiden, The Netherlands.

Cell
|March 20, 2012
PubMed
概括

拼接的失调,一个改变基因表达的过程,导致许多疾病. 治疗策略正在出现,以纠正拼接错误,为遗传和获得的疾病提供新的治疗可能性.

科学领域:

  • 分子生物学分子生物学
  • 遗传学 是一个遗传学.
  • 治疗方法 治疗方法

背景情况:

  • 拼接和替代拼接是关键的基因表达过程.
  • 这些过程的失调与许多遗传和获得性疾病有关.
  • 定位拼接提供了一个有前途的治疗途径.

研究的目的:

  • 提供对调节拼接的最新治疗策略的概述.
  • 突出在临床和临床前环境中的成功.
  • 通过针对拼接来讨论产生有益蛋白质和去除有害蛋白质的方法.

主要方法:

  • 关于拼接调制疗法的最新文献的综述.
  • 对临床和临床前数据的分析.
  • 基于其作用机制的治疗策略的分类.

主要成果:

  • 有几种策略可以有效地调节剪接的治疗效果.
  • 方法包括恢复开放的阅读框架,影响替代拼接,并诱导外子纳入.
  • 这些方法旨在产生功能性蛋白质或消除有害的蛋白质.

结论:

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  • 治疗拼接调制是一个快速发展的领域.
  • 成功的策略正在转化为临床和临床前环境.
  • 针对性拼接具有治疗广泛疾病的巨大潜力.