CRISPR-CasRx-mediated disruption of Aqp1/Adrb2/Rock1/Rock2 genes reduces intraocular pressure and retinal ganglion cell damage in mice
Brief intro:
- Author: Mingyu Yao, Zhenhai Zeng, Siheng Li, Zhilin Zou, Zhongxing Chen, Xinyi Chen, Qingyi Gao, Guoli Zhao, Aodong Chen, Zheng Li, Yiran Wang, Rui Ning, Colm McAlinden, Xingtao Zhou & Jinhai Huang
- Journal: Nature Communications
- Doi: https://www.doi.org/10.1038/s41467-024-50050-4
- Publication Date: 2024 Jul 30
Products/Services used in the paper
Quotation shows PackGene:All AAV used in this study were packaged by PackGene Biotech (Guangzhou, China).
Research Field:retinal ganglion cell damage
AAV Serotype:AAV2, ShH10
Targeted organ:eye
Animal or cell line strain:mice
Abstract
Glaucoma affects approximately 80 million individuals worldwide, a condition for which current treatment options are inadequate. The primary risk factor for glaucoma is elevated intraocular pressure. Intraocular pressure is determined by the balance between the secretion and outflow of aqueous humor. Here we show that using the RNA interference tool CasRx based on shH10 adenovirus-associated virus can reduce the expression of the aqueous humor circulation related genes Rock1 and Rock2, as well as aquaporin 1 and β2 adrenergic receptor in female mice. This significantly reduced intraocular pressure in female mice and provided protection to the retina ganglion cells, ultimately delaying disease progression. In addition, we elucidated the mechanisms by which the knockdown of Rock1 and Rock2, or aquaporin 1 and β2 adrenergic receptor in female mice, reduces the intraocular pressure and secures the retina ganglion cells by single-cell sequencing.
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