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聚焦超声波介导的大脑基因组编辑

Yeh-Hsing Lao1,2, Robin Ji1, Joyce K Zhou1

  • 1Department of Biomedical Engineering, Columbia University, New York, NY 10027.

Proceedings of the National Academy of Sciences of the United States of America
|August 14, 2023
PubMed
概括

聚焦超声波 (FUS) 可以安全地打开血脑屏障,用于基因编辑. 这种非侵入性方法可以通过静脉注射提供CRISPR/Cas9基因编辑工具,克服了以前用于大脑治疗的交付挑战.

关键词:
这就是CRISPR/Cas9的作用.血脑屏障 血脑屏障 血脑屏障 血脑屏障聚焦式超声波的使用基因传递 基因传递 基因传递基因组编辑 基因组编辑

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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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