A locked nucleic acid antisense oligonucleotide (LNA) silences PCSK9 and enhances LDLR expression in vitro and in vivo

Nidhi Gupta, Niels Fisker, Marie-Claude Asselin, Marie Lindholm, Christoph Rosenbohm, Henrik Ørum, Joacim Elmén, Nabil G Seidah, Ellen Marie Straarup

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Abstrakt

The proprotein convertase subtilisin/kexin type 9 (PCSK9) is an important factor in the etiology of familial hypercholesterolemia (FH) and is also an attractive therapeutic target to reduce low density lipoprotein (LDL) cholesterol. PCSK9 accelerates the degradation of hepatic low density lipoprotein receptor (LDLR) and low levels of hepatic PCSK9 activity are associated with reduced levels of circulating LDL-cholesterol.
OriginalsprogEngelsk
Artikelnummere10682
TidsskriftP L o S One
Vol/bind5
Udgave nummer5
ISSN1932-6203
DOI
StatusUdgivet - 2010
Udgivet eksterntJa

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