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US 9,434,947 B2Granted
Modulation of KCNH2 isoform expression by oligonucleotides as a therapeutic approach for long QT syndrome
AssigneeOregon Health & Science University
InventorsZhengfeng Zhou, Qiuming Gong, Matthew Stump
Life of this patent
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Abstract
Oligonucleotides with activity in preventing poly(A) adenylation at intron 9 of the KCNH2 gene, as well as pharmaceutical compositions comprising the oligonucleotides and methods of using the oligonucleotides to treat long QT syndrome in a subject are disclosed. The oligonucleotides include antisense sequences corresponding to sites termed DSE-1 and DSE-2 in intron 9.
Claims7
An isolated oligonucleotide comprising SEQ ID NO:
- 4 that inhibits the formation of a poly(A) signal in intron 9 of KCNH2, wherein the oligonucleotide is no more than 30 nucleotides in length and wherein the oligonucleotide comprises a modified nucleotide, a locked nucleotide, a G-clamp nucleotide, a nucleotide base analog, a 3′-terminal cap moiety, or a phosphate backbone modification.
Dependencies are inferred from the claim text.