Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies
AssigneeRogCon, Inc.
InventorSteven Petrou
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Abstract
Provided herein are methods, compounds, and compositions for reducing expression of SCN2A in a subject. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate an SCN1A related disease or disorder (e.g., Dravet syndrome) in a subject in need.
Claims27
A method of reducing seizures in a subject with an SCN1A encephalopathy, the method comprising administering to the subject a compound comprising a single-stranded oligonucleotide that 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.
Dependencies are inferred from the claim text.