Patent ID: 11939582
Assignee: ROGCON, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A method of treating an SCN1A encephalopathy in a subject in need thereof, the method comprising administering to the subject a composition comprising a single-stranded oligonucleotide, wherein the single-stranded oligonucleotide is 10-80 nucleosides in length and has a nucleobase sequence comprising a portion of 10 contiguous nucleobases having at least 80% complementarity to an equal length portion of a target region of a pre-mRNA transcript or an mRNA transcript of a human SCN2A gene, in an amount and for a duration sufficient to treat the SCN1A encephalopathy, wherein the subject has a loss-of-function mutation in SCN1A, and wherein the single-stranded oligonucleotide is selective for SCN2A pre-mRNA or mRNA over SCN1A pre-mRNA or mRNA.