医学
家族性高胆固醇血症
核酸
疾病
遗传增强
冠心病
DNA
生物信息学
胆固醇
基因
内科学
遗传学
生物
作者
Tulsi Ram Damase,Roman Sukhovershin,Biana Godin,Khurram Nasir,John P. Cooke
出处
期刊:Circulation
[Ovid Technologies (Wolters Kluwer)]
日期:2024-08-27
卷期号:150 (9): 724-735
标识
DOI:10.1161/circulationaha.123.067957
摘要
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.
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