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Updated: Feb 9, 2026

Author Spotlight: Intrathecal Injection – An Efficient and Reliable Delivery Method to Test the Efficacy of Gene Editing in Neonatal Mouse Brains
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Author Spotlight: Intrathecal Injection – An Efficient and Reliable Delivery Method to Test the Efficacy of Gene Editing in Neonatal Mouse Brains

Published on: March 8, 2024

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一种组合合成策略,用于开发基因组编辑蛋白递送剂,针对小鼠视网膜

Jianye Zhang1, Rafał Hołubowicz1,2, Roman Smidak1

  • 1Gavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.

Nature communications
|February 7, 2026
PubMed
概括

来自库马西蓝 (CBB) 的新脂类物质有效地将基因编辑蛋白质输送到眼睛. 这一突破推动了对遗传视网膜疾病 (IRD) 的CRISPR/Cas9疗法,为单剂量治疗提供了潜力.

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