开发和应用CRISPR-Cas9用于基因组工程
Patrick D Hsu1, Eric S Lander2, Feng Zhang3
1Broad Institute of MIT and Harvard, 7 Cambridge Center, Cambridge, MA 02141, USA; McGovern Institute for Brain Research, Department of Brain and Cognitive Sciences, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA; Department of Molecular and Cellular Biology, Harvard University, Cambridge, MA 02138, USA.
Cell
|June 7, 2014
概括
克里斯普尔-Cas9基因编辑技术允许科学家精确修改DNA序列. 这种强大的工具使研究人员能够研究基因组功能并了解遗传变异.
科学领域:
- 基因组学就是基因组学.
- 分子生物学分子生物学
- 生物技术是生物技术.
背景情况:
- 与CRISPR相关的 (Cas) 系统,特别是Cas9,是微生物的防御机制.
- 这些系统利用RNA引导的内核酶进行向的DNA操纵.
- 最近的进展使得对哺乳动物基因组功能的系统调查成为可能.
研究的目的:
- 审查Cas9技术的发展和应用.
- 突出Cas9在基因组工程和功能基因组学中的作用.
- 讨论基于Cas9的研究中的挑战和未来方向.
主要方法:
- Cas9由RNA引导到特定的基因组位置.
- 在内源基因组中编辑或调节DNA序列.
- 可扩展的遗传干扰,用于系统级分析.
主要成果:
- Cas9可以在各种生物体中精确编辑DNA.
- 有助于阐明基因组的组织和功能.
- 建立遗传变异和表型之间的因果关系.
结论:
- Cas9技术正在彻底改变基础生物学,生物技术和医学.
- 它的简单性和可扩展性使得全面的基因组研究成为可能.
- 目前正在进行的研究有望进一步创新应用.
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