MODULATION OF KCNH2 ISOFORM EXPRESSION BY OLIGONUCLEOTIDES AS A THERAPEUTIC APPROACH FOR LONG QT SYNDROME

摘要

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.

主权项

1. An oligonucleotide comprising SEQ ID NO: 3 that inhibits formation of a poly(A) signal in intron 9 of KCNH2, wherein the oligonucleotide is no more than 30 nucleotides in length