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US 9,957,508 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.
Claims13
An oligonucleotide no more than 30 nucleotides in length, comprising SEQ ID NO:
- 3 and at least one of a modified nucleotide, locked nucleotide, G-clamp nucleotide, nucleotide base analog, 3′-terminal cap moiety, or phosphate backbone modification, provided that the oligonucleotide inhibits formation of a poly(A) signal in intron 9 of KCNH2.
Dependencies are inferred from the claim text.