Transgene expression of Stanniocalcin-1 provides sustained intraocular pressure reduction by increasing outflow facility.
Transgene expression of Stanniocalcin-1 provides sustained intraocular pressure reduction by increasing outflow facility.
复制标题
Stanniocalcin-1的转基因表达通过增加流出设施，可提供持续的眼内压降。
DOI：
10.1371/journal.pone.0269261
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发表时间：
2022
期刊：
影响因子：
3.7
通讯作者：
中科院分区：
文献类型：
--
作者：
青光眼是世界范围内不可逆性失明的主要原因。青光眼的治疗是针对降低眼内压（IOP），这是主要的风险因素，也是唯一可靠的治疗目标，通过局部药物或手术干预，包括激光或手术。虽然局部治疗通常是第一线，但只有不到50%的患者按规定服用滴剂。正在研究延长时间段降低IOP的持续释放技术用于临床使用。我们最近确定了斯钙素-1，一种天然存在的激素，作为降低IOP的药物。在这里，我们表明，一个单一的注射到前房的小鼠与腺相关病毒载体含有斯钙素-1的转基因的结果在弥漫性和持续表达的蛋白质，并产生IOP降低长达6个月。随着治疗效果开始减弱，可以通过重复注射来挽救降低IOP。房水动力学研究表明，增加外流设施的行动机制。这种一流的治疗方法有可能改善目前全球8000万青光眼患者的护理并降低视力丧失率。
Glaucoma is the leading cause of irreversible blindness worldwide. Therapies for glaucoma are directed toward reducing intraocular pressure (IOP), the leading risk factor and only reliable therapeutic target via topical medications or with procedural intervention including laser or surgery. Though topical therapeutics are typically first line, less than 50% of patients take drops as prescribed. Sustained release technologies that decrease IOP for extended periods of time are being examined for clinical use. We recently identified Stanniocalcin-1, a naturally occurring hormone, as an IOP-lowering agent. Here, we show that a single injection into the anterior chamber of mice with an adeno-associated viral vector containing the transgene of stanniocalcin-1 results in diffuse and sustained expression of the protein and produces IOP reduction for up to 6 months. As the treatment effect begins to wane, IOP-lowering can be rescued with a repeat injection. Aqueous humor dynamic studies revealed an increase in outflow facility as the mechanism of action. This first-in-class therapeutic approach has the potential to improve care and reduce the rates of vision loss in the 80 million people worldwide currently affected by glaucoma.
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影响因子：
1.7
作者：
Baudouin, Christophe;Renard, Jean-Paul;Bouee, Stephane
通讯作者：
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3.7
作者：
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DOI：
10.1016/j.bbrc.2014.09.060
发表时间：
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影响因子：
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作者：
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Semba, Charles P.