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Blood-brain barrier–penetrating single CRISPR-Cas9 nanocapsules for effective and safe glioblastoma gene therapy

Yan Zou#, Xinhong Sun#, Qingshan Yang, Meng Zheng*, Olga Shimoni, Weimin Ruan, Yibin Wang, Dongya Zhang, Jinlong Yin, Xiangang Huang, Wei Tao, Jong Bae Park, Xing-Jie Liang, Kam W. Leong, Bingyang Shi*

Science Advances 2022, 8, eabm8011 (IF=14.98)

Abstract

We designed a unique nanocapsule for efficient single CRISPR-Cas9 capsuling, noninvasive brain delivery and tumor cell targeting, demonstrating an effective and safe strategy for glioblastoma gene therapy. Our CRISPR-Cas9 nanocapsules can be simply fabricated by encapsulating the single Cas9/sgRNA complex within a glutathione-sensitive polymer shell incorporating a dual-action ligand that facilitates BBB penetration, tumor cell targeting, and Cas9/sgRNA selective release. Our encapsulating nanocapsules evidenced promising glioblastoma tissue targeting that led to high PLK1 gene editing efficiency in a brain tumor (up to 38.1%) with negligible (less than 0.5%) off-target gene editing in high-risk tissues. Treatment with nanocapsules extended median survival time (68 days versus 24 days in nonfunctional sgRNA-treated mice). Our new CRISPR-Cas9 delivery system thus addresses various delivery challenges to demonstrate safe and tumor-specific delivery of gene editing Cas9 ribonucleoprotein for improved glioblastoma treatment that may potentially be therapeutically useful in other brain diseases.


该论文的相关报道:

New Scientist: https://www.newscientist.com/article/2316878-crispr-nanocapsule-limits-growth-of-aggressive-brain-tumours-in-mice/

生物世界: https://mp.weixin.qq.com/s/46bReHIKKXZN652g8_FDSA



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