Summary
The current technology consists for compositions under development for the treatment of cardiovascular arrhythmias, specifically Long-QT Syndrome.
Technology Overview
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), a disease associated with prolonged cardiac action potential duration and prolonged QT intervals on ECG, which can lead to ventricular arrhythmia and sudden death. The current morpholino oligonucleotides upregulate the Kv11.1 subunit of the hERG gene to treat long-QT syndrome caused by mutations.
Publication
Gong Q, et al., “Upregulation of functional Kv11.1 isoform expression by inhibition of intronic polyadenylation with antisense morpholino oligonucleotides.” J Mol Cell Cardiol. 2014 Nov;76:26-32. Link
Licensing Opportunity
This technology is available for licensing.