Oligonucleotides for Long QT Syndrome
OHSU # 2044
Oligonucleotides to prevent poly(A) adenylation at intron 9 of the KCNH2 gene, and pharmaceutical compositions comprising the oligonucleotides and methods to use same to treat long QT syndrome. Included are antisense sequences to specific sites in intron 9.
KCNH2, also known as ether-a-go-go related gene 1 (hERG1) encodes the Kv11.1 channel that conducts the rapidly activating delayed rectifier K+ current in the heart. These channels are essential for cardiac action potential repolarization. Mutations in KCNH2 cause long QT syndrome type 2 (LQT2). The OHSU oligonucleotides are morpholino oligonucleotides which can be used to treat long-QT syndrome caused by mutations in the KCNH2 gene.
US Non-Provisional and PCT patent application filed 01/20/2015.
US Patent 9,434,947 issued Sept. 06, 2016.
|Published||Patent Cooperation Treaty||WO 2016/118118|
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