Antisense Nucleic Acids for Use in the Treatment for KCNQ1 Mutation Carriers

Abstract: the invention relates to isolated antisense molecule capable of inhibiting the expression of the kcnq1 gene in a mammalian cell, wherein said antisense molecule comprises an anti-sense nucleic acid strand which is substantially complementary to a target region of a transcript encoded by the kcnq1 gene, wherein said antisense nucleic acid strand is at least complementary to snp rs1057128, rs8234 or rs17215465 in said target region.

Inventor(s): Yigal-Martin Pinto, Anke Johanna Marina Tijsen, Lucia Cocera Ortega

CPC Classification: C12N15/1138 ({against receptors or cell surface proteins})

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