Invention Grant
US09434947B2 Modulation of KCNH2 isoform expression by oligonucleotides as a therapeutic approach for long QT syndrome 有权
通过寡核苷酸调节KCNH2同种型表达作为长QT综合征的治疗方法

Modulation of KCNH2 isoform expression by oligonucleotides as a therapeutic approach for long QT syndrome
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.
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