Zusammenfassung
Proproteinkonvertase Subtilisin/Kexin-Typ 9 (PCSK9) reguliert durch Degradation des LDL-Rezeptors das Serum-LDL-Cholesterin. „Loss-of-function“-PCSK9-Mutationen resultieren in sehr niedrigen LDL-Cholesterin-Serumspiegeln und schützen vor kardiovaskulären Erkrankungen, wohingegen „Gain-of-function“-Mutationen das Serum-LDL-Cholesterin erhöhen. In-vitro- und In-vivo-Experimente zeigen, dass Antikörper gegen PCSK9 eine neue Therapieoption bei kardiovaskulären Patienten darstellen könnten. Dieser Beitrag fasst die Biochemie und die Funktion von PCSK9 sowie die Ergebnisse kürzlich publizierter Phase-II-Studien zusammen.
Abstract
Proprotein convertase subtilisin/kexin type 9 (PCSK 9) is a key regulator of cholesterol homeostasis acting via degradation of the low density lipoprotein (LDL) receptor. Loss of function PCSK 9 mutations result in very low LDL cholesterol serum levels and protection from cardiovascular disease whereas gain of function mutations increase serum LDL cholesterol. Based on in vitro and in vivo data antibodies targeting PCSK 9 have now emerged as a novel treatment option in patients with cardiovascular disease. This review briefly summarizes the biochemistry and function of PCSK9 and the results from recent phase II trials.
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Interessenkonflikt. P. Stawowy und E. Fleck geben an, klinische Studien mit Amgen durchzuführen. S. Kelle gibt an, dass kein Interessenkonflikt besteht.
Dieser Beitrag beinhaltet keine Studien an Menschen oder Tieren.
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Stawowy, P., Kelle, S. & Fleck, E. PCSK9 als neues Target in der Therapie der Hypercholesterinämie. Herz 39, 466–469 (2014). https://doi.org/10.1007/s00059-013-3913-0
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DOI: https://doi.org/10.1007/s00059-013-3913-0