Hegele Robert A
Departments of Medicine, (Division of Endocrinology) and Biochemistry, Western University, London, Canada.
touchREV Endocrinol. 2022 Nov;18(2):82-83. doi: 10.17925/EE.2022.18.2.82. Epub 2022 Nov 2.
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- agents, such as the short interfering RNA ARO-APOC3, represents a promising approach to correct the severe biochemical disturbance in FCS.
患有家族性乳糜微粒血症综合征（FCS）的患者由于遗传上缺乏脂解能力而患有严重的高甘油三酯血症。他们对传统疗法反应不佳。为降低潜在致命性胰腺炎的风险，FCS的管理主要依赖严格的低脂饮食，但这种饮食难以遵循且会影响生活质量。使用新型抗剂，如短干扰RNA ARO-APOC3，针对性降低载脂蛋白C-III，是纠正FCS严重生化紊乱的一种有前景的方法。
