家族性乳糜小贫血综合征的APOC3干扰。

Robert A Hegele
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引用次数: 3

摘要

家族性乳糜小血症综合征(FCS)患者有严重的高甘油三酯血症,这是由于遗传上缺乏溶脂能力。他们对传统疗法的反应很差。为了降低潜在致命性胰腺炎的风险,FCS的管理主要依赖于严格的低脂饮食,这很难遵循并影响生活质量。使用新的抗apoc3药物(如短干扰RNA ARO-APOC3)靶向减少载脂蛋白C-III,是纠正FCS严重生化干扰的一种有希望的方法。
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APOC3 Interference for Familial Chylomicronaemia Syndrome.

Patients with familial chylomicronaemia syndrome (FCS) have severe hypertriglyceridaemia due to genetically absent lipolytic capacity. They have a poor response to conventional therapies. To reduce the risk of potentially fatal pancreatitis, the management of FCS relies principally on a strict low-fat diet, which is difficult to follow and compromises quality of life. Targeted reduction of apolipoprotein C-III using new anti-APOC3 agents, such as the short interfering RNA ARO-APOC3, represents a promising approach to correct the severe biochemical disturbance in FCS.

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