Fewer jabs, decreased cost: Gaucher disease is caused by a deficiency in β-glucocerebrosidase (GCase). Although enzyme replacement therapy can be applied using mannose-exposed conjugated GCase, the low stability of the enzyme in blood demands periodic intravenous administration, adding to the high cost of treatment. In this work, GCase was encapsulated inside virus-like nanoparticles, and their surface was functionalized with mannose groups for targeting to macrophages. These nanoreactors showed significant GCase activity. Importantly, the enzymes were found to be 11-fold more stable under physiological conditions after encapsulation, which could decrease the cost of therapy by reducing the number of required intravenous injections.

中文翻译：

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具有 β-葡萄糖脑苷脂酶活性的靶向酶 VLP 纳米反应器作为戈谢病的潜在酶替代疗法

减少注射次数，降低成本：戈谢病是由 β-葡萄糖脑苷脂酶 (GCase) 缺乏引起的。虽然可以使用暴露于甘露糖的缀合GCase进行酶替代疗法，但酶在血液中的稳定性较低，需要定期静脉注射，从而增加了治疗成本。在这项工作中，GCase 被封装在病毒样纳米粒子内，其表面用甘露糖基团进行功能​​化，以靶向巨噬细胞。这些纳米反应器表现出显着的 GCase 活性。重要的是，这些酶在封装后在生理条件下稳定性提高了 11 倍，这可以通过减少所需的静脉注射次数来降低治疗成本。