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相关概念视频

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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RNA Splicing01:32

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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...
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CRISPR and crRNAs02:53

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
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通过双重CRISPR引导的3'转接拼接重写内源人类转录.

Sita S Chandrasekaran1, Cyrus Tau2, Becky Xu Hua Fu3

  • 1Arc Institute, 3181 Porter Drive, Palo Alto, CA 94304, USA; Department of Bioengineering, University of California, Berkeley, Berkeley, CA, USA; University of California, Berkeley, San Francisco Graduate Program in Bioengineering, Berkeley, CA, USA.

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概括

研究人员开发了使用Cas编辑器 (RESPLICE) 的RNA引导转接,用于暂时RNA编辑. 这种方法有效地和具体地将RNA载荷插入内源转录中,以最小的目标外影响精确控制细胞功能.

关键词:
克里斯普尔是什么意思?克里斯普尔是什么意思?在Cas13中,Cas13是Cas13中的一个.这就是Cas7-11的原因.编辑RNA的RNA编辑替代性拼接是一种替代性的拼接.嵌合式RNAs 是一种化学RNA.埃克森替代品的替代品分子工程 分子工程是指分子工程.合成生物学 合成生物学通过拼接进行跨拼接.

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科学领域:

  • 分子生物学分子生物学
  • 基因编辑技术的技术
  • 在RNA治疗方面,RNA疗法.

背景情况:

  • 与基因组编辑相比,RNA编辑提供了短暂的细胞改变,具有较低的目标外风险.
  • 现有的跳过外体的技术主要影响到拼接地点的选择.
  • 许多转录组的修改需要外源的外子添加或替换治疗应用.

研究的目的:

  • 开发一种新的RNA编辑技术,用于精确的转录组修改.
  • 为了使外源性外子能够被替换或添加到目标信使RNA (mRNA) 中.
  • 通过RNA操纵对细胞功能进行临时和可编程的控制.

主要方法:

  • 使用Cas编辑器 (RESPLICE) 开发RNA引导的转接.
  • 利用两个正交的RNA向CRISPR效应器进行同局部化和cis-splicing抑制.
  • 在多种细胞类型的内源转录中展示了RNA载荷插入.

主要成果:

  • 实现了RNA货物的高效,特定和可编程的转链拼接 (高达2.1kb).
  • 在3种不同的细胞类型中成功准了11种内源性转录.
  • 在批量种群中显示高达45%的转接效率,在高效应表达的排序细胞中显示高达90%.

结论:

  • 复制代表了一种新的RNA编辑模式.
  • 这项技术使得能够精心调整和暂时控制蜂程序.
  • RESPLICE为基于RNA的治疗策略和生物研究提供了一个有前途的工具.