他汀类药物、依折麦布和PCSK9抑制剂的出现极大改善了家族性高胆固醇血症（FH）的预后。然而，尽管进行了最大限度的降脂治疗，许多FH患者的低密度脂蛋白胆固醇（LDL-C）水平仍达不到指南推荐的水平。不依赖于低密度脂蛋白（LDL）受体活性而降低LDL的 ...

该疗法采用 CRISPR-Cas9 和两个向导RNA，靶向HIV基因组中的三个位点 ... 递送腺嘌呤碱基编辑器的mRNA和靶向PCSK9基因的 gRNA。这种碱基编辑技术能够在不依赖DNA双链断裂的情况下，实现对单个碱基的定向修改，从而永久关闭 PCSK9 基因的表达（图8）。

PCSK9抑制剂的使用正在为临床上难治性高胆固醇血症以及他汀类药物不耐受的患者群体开辟全新的治疗前景。 1月10日，恒瑞医药的一款抗PCSK9药物瑞 ...

Cardiovascular diseases remain the most common cause of death worldwide (Roth et al., 2020). Although new therapies have been developed in recent decades, there is still an urgent need for new ...

Utilizing the CRISPR-Cas9 system, this approach eliminates the ... is an in-vivo liver gene-editing therapy targeting the PCSK9 gene to reduce LDL cholesterol production by disrupting PCSK9 ...

Among the most exciting breakthroughs are gene therapy and gene editing, with CRISPR-Cas9 leading the charge. This remarkable tool, inspired by bacteria's natural defenses, acts like precise ...