Despite the intensive research and progress in modern pharmacotherapy, hypercholesterolemia
and related cardiovascular complications remain one of the leading causes of mortality and
disability in the modern world. A significant contribution to the treatment of hypercholesterolemia
was made by the discovery of proprotein convertase subtilisin/kexin type 9 (PCSK9). This enzyme is
responsible for the degradation of the low-density lipoprotein (LDL) receptor (LDLR) found at the
surface of the plasma membrane in the liver and directly associated with serum LDL level. Limitations
in standard therapy used in the treatment of lipid disorders have led to the development of new drugs,
such as an inhibitor of PCSK9. Over the past years, the greatest achievement in discovering the
PCSK9 inhibitor was made by designing monoclonal antibodies that disable PCSK9 to bind LDLR
and RNA interference to reduce PCSK9 production, but one of the main disadvantages is costeffectiveness.
In this review, we will summarize the most recent findings of basic and clinical studies
which focus on PCSK9 function, regulation and therapeutic target for the treatment of hypercholesterolemia
and associated cardiovascular diseases.
Keywords: PCSK9, PCSK9 and hypercholesterolemia, PCSK9 and CVD, PCSK9 therapy, evolocumab, alirocumab.
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