Familial hypercholesterolemia (FH) is a genetic disease that leads to elevated low-density lipoprotein cholesterol levels and risk of coronary heart disease. Current therapeutic options for FH remain relatively limited and only partially effective in both lowering low-density lipoprotein cholesterol and modifying coronary heart disease risk. The unique characteristics of nucleic acid therapies to target the underlying cause of the disease can offer solutions unachievable with conventional medications. DNA- and RNA-based therapeutics have the potential to transform the care of patients with FH. Recent advances are overcoming obstacles to clinical translation of nucleic acid-based medications, including greater stability of the formulations as well as site-specific delivery, making gene-based therapy for FH an alternative approach for treatment of FH.

中文翻译：

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针对家族性高胆固醇血症的已建立和新兴核酸疗法。


家族性高胆固醇血症（FH）是一种遗传性疾病，会导致低密度脂蛋白胆固醇水平升高和冠心病风险。目前 FH 的治疗选择仍然相对有限，并且在降低低密度脂蛋白胆固醇和改变冠心病风险方面仅部分有效。核酸疗法针对疾病根本原因的独特特性可以提供传统药物无法实现的解决方案。基于 DNA 和 RNA 的疗法有可能改变 FH 患者的护理。最近的进展正在克服基于核酸的药物临床转化的障碍，包括更高的制剂稳定性以及位点特异性递送，使得基于基因的 FH 治疗成为 FH 治疗的替代方法。