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相关概念视频

In-vitro Mutagenesis01:16

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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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相关实验视频

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Engineering and Evolution of Synthetic Adeno-Associated Virus AAV Gene Therapy Vectors via DNA Family Shuffling
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使用单个AAV载体的替代基因编辑策略

Jamie C Moffa1,2, Vani Kalyanaraman1, Bryan A Copits1

  • 1Washington University Pain Center, Department of Anesthesiology, Washington University School of Medicine, St. Louis, MO, USA.

Bio-protocol
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概括
此摘要是机器生成的。

本研究介绍了一种单向量CRISPR/Cas9基因编辑和使用腺相关病毒 (AAV) 进行转基因表达的协议. 这种方法可以在小鼠神经系统中进行特定细胞类型的编辑和工具传递.

关键词:
这就是CRISPR/Cas9的作用.基因编辑 基因编辑影像成像技术 影像成像技术光学遗传学和光学遗传学.摄影测量摄影仪的使用工具 工具 工具 工具

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Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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科学领域:

  • 神经科学是一个神经科学.
  • 分子生物学分子生物学
  • 遗传学 是一个遗传学.

背景情况:

  • 通过CRISPR/Cas9基因编辑,可以精确地修改DNA.
  • 腺相关病毒 (AAV) 是神经系统中基因传递的有效载体.
  • 在特定细胞类型中同时进行基因编辑和转基因表达具有挑战性.

研究的目的:

  • 提出设计和生成单向量等离子体和AAV用于特定细胞类型的CRISPR/Cas9编辑和转基因表达的协议.
  • 为了使编辑细胞中的标准神经科学工具能够共同表达.
  • 促进研究中枢神经系统和外围神经系统中的基因功能.

主要方法:

  • 指导RNA (gRNA) 设计用于针对特定的基因.
  • 结合和克隆具有CRISPR能力的AAV载体.
  • AAVs的生产和病毒标位量化.
  • 与Cre依赖的Cas9小鼠线路的兼容性,用于选择性协同表达.

主要成果:

  • 描述了一个生成单向量CRISPR/Cas9兼容的AAV的协议.
  • AAVs可以在任何血清型中生产,以实现多样化的分娩.
  • 该系统允许细胞类型特定的编辑和共表达工具,如光蛋白,光遗传学和化学遗传学工具.
  • 这些载体适用于中枢神经系统和外围神经系统.

结论:

  • 这种单向量方法简化了在小鼠神经系统中的基因编辑和工具交付.
  • 该协议为研究神经回路中的基因功能提供了一种灵活的方法.
  • 这种技术可以加速发现和功能测试涉及突触传输,电路活动和形态学的新基因.