PDF(Pubmed)
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.
摘要：
青光眼影响全球约8000万人，目前治疗方案不足的情况。青光眼的主要危险因素是眼内压升高。眼内压由房水的分泌和流出之间的平衡决定。在这里，我们表明使用基于shH10腺病毒相关病毒的RNA干扰工具CasRx可以降低雌性小鼠房水循环相关基因Rock1和Rock2以及水通道蛋白1和β2肾上腺素能受体的表达。这显着降低了雌性小鼠的眼内压，并为视网膜神经节细胞提供了保护，最终延缓疾病进展。此外,我们阐明了雌性小鼠中Rock1和Rock2或水通道蛋白1和β2肾上腺素能受体敲低的机制，通过单细胞测序降低眼内压并确保视网膜神经节细胞。
