基因编辑CRISPR 纠正TGFBI突变在自体主导角膜变的基因编辑

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Updated: Feb 28, 2026

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

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Jue Chen1, Connor W Davison2,3, James Ellis2

  • 1Department of Biological Chemistry, University of Michigan, Ann Arbor, Michigan, United States.

Investigative ophthalmology & visual science
|February 27, 2026
PubMed
概括

这项研究开发了一种新的腺基编辑器 (ABE) 疗法,以纠正常见的TGFBI突变,导致角膜发育不良. 在临床前模型中,ABE方法显示出高效率和安全性,提供了一个有前途的新治疗策略.

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