Tulsi R Damase, Roman Sukhovershin, Biana Godin, Khurram Nasir, John P Cooke
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引用次数: 0
摘要
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增加。目前针对家族性高胆固醇血症的治疗方案仍然相对有限,而且只能部分有效地降低低密度脂蛋白胆固醇和改变冠心病风险。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 的疗法有可能改变对 FH 患者的治疗。最近的进展正在克服核酸类药物临床转化的障碍,包括提高制剂的稳定性和特异性给药,使FH的基因疗法成为治疗FH的另一种方法。
Established and Emerging Nucleic Acid Therapies for Familial Hypercholesterolemia.
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.
期刊介绍:
Circulation is a platform that publishes a diverse range of content related to cardiovascular health and disease. This includes original research manuscripts, review articles, and other contributions spanning observational studies, clinical trials, epidemiology, health services, outcomes studies, and advancements in basic and translational research. The journal serves as a vital resource for professionals and researchers in the field of cardiovascular health, providing a comprehensive platform for disseminating knowledge and fostering advancements in the understanding and management of cardiovascular issues.