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AssigneeProQR Therapeutics II B.V.
InventorsGerardus Johannes PLATENBURG, Bart KLEIN, Lenka VAN SINT FIET, Maarten HOLKERS
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The invention relates to guide oligonucleotides that can bring about specific changes to a target RNA or DNA molecule in a eukaryotic cell, preferably liver cells, for use in the treatment of hypercholesterolemia, cardiovascular disease, liver injury and/or alcohol-induced steatohepatitis in human subjects. More specifically, the invention relates to guide oligonucleotides and the use thereof in the editing of nucleic acids encoding an auto-cleavage site within the PCSK9 proprotein, thereby inhibiting, or inactivating the PCSK9 protein (or the matured PCSK9 complex) in its ability to cause LDL receptor protein degradation.
A guide oligonucleotide capable of inducing editing of a nucleic acid encoding a human Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) proprotein, wherein the nucleic acid editing decreases or prevents the ability of the PCSK9 proprotein from being processed by auto-cleavage of a proteolytic cleavage site.
Dependencies are inferred from the claim text.