Patent ID: 11873490
Assignee: STOKE THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A method of treating a human subject in need thereof, the method comprising administering to the human subject a therapeutic agent or a vector encoding the therapeutic agent, wherein the therapeutic agent modulates splicing of a non-sense mediated decay-inducing exon (NMD exon) from a pre-mRNA that encodes Nav1.1 protein in a cell of the human subject, wherein the therapeutic agent binds to a targeted portion of a pre-mRNA that contains the NMD exon and that encodes Nav1.1 protein, and wherein the targeted portion (i) is within an intron sequence flanking the NMD exon; (ii) comprises at least one nucleotide of the NMD exon; or (iii) is within the NMD exon.