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C3 Transferase-Expressing scAAV2 Transduces Ocular Anterior Segment Tissues and Lowers Intraocular Pressure in Mouse and Monkey.
Molecular Therapy - Methods & Clinical Development ( IF 4.6 ) Pub Date : 2019-11-29 , DOI: 10.1016/j.omtm.2019.11.017
Junkai Tan 1 , Xizhen Wang 2 , Suping Cai 2 , Fen He 2 , Daren Zhang 1 , Dongkan Li 1 , Xianjun Zhu 3, 4 , Liang Zhou 4 , Ning Fan 2 , Xuyang Liu 1, 2
Affiliation  

Glaucoma is a lifelong disease with elevated intraocular pressure (IOP) as the main risk factor, and reduction of IOP remains the major treatment for this disease. However, current IOP-lowering therapies are far from being satisfactory. We have demonstrated that the lentivirus-mediated exoenzyme C3 transferase (C3) expression in rat and monkey eyes induced relatively long-term IOP reduction. We now show that intracameral injection of self-complementary AAV2 containing a C3 gene into mouse and monkey eyes resulted in morphological changes in trabecular meshwork and IOP reduction. The vector-transduced corneal endothelium and the C3 transgene expression, not vector itself, induced corneal edema as a result of actin-associated endothelial barrier disruption. There was a positive (quadratic) correlation between measured IOP and grade of corneal edema. This is the first report of using an AAV to transduce the trabecular meshwork of monkeys with a gene capable of altering cellular structure and physiology, indicating a potential gene therapy for glaucoma.

中文翻译:

表达C3转移酶的scAAV2转导小鼠和猴子的眼前节组织并降低眼内压。

青光眼是一种终生疾病,其眼内压升高(IOP)是主要危险因素,而降低IOP仍是该疾病的主要治疗方法。但是,目前降低眼压的疗法远不能令人满意。我们已经证明,慢病毒介导的外切酶C3转移酶(C3)在大鼠和猴眼中的表达诱导了相对长期的IOP降低。我们现在显示向小鼠和猴眼前房内注射含有C3基因的自互补AAV2会导致小梁网和IOP减少的形态变化。由于肌动蛋白相关的内皮屏障破坏,载体转导的角膜内皮和C3转基因表达(而不是载体本身)诱导角膜水肿。测得的眼压和角膜浮肿程度之间存在正相关(二次相关)。
更新日期:2019-11-29
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