Compounds and methods for reducing kcnt1 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsHuynh-Hoa Bui, Susan M. Freier
Life of this patent
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Abstract
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of KCNT1 RNA in a cell or subject, and in certain instances reducing the amount of KCNT1 protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurological condition. Such symptoms and hallmarks include seizures, encephalopathy, and behavioral abnormalities. Non-limiting examples of neurological conditions that benefit from these compounds, methods, and pharmaceutical compositions are epilepsy of infancy with migrating focal seizures (EIMFS), autosomal dominant nocturnal frontal lobe epilepsy (ADNFLE), West syndrome, and Ohtahara syndrome.
Claims41
-54. (canceled)
A modified oligonucleotide consisting of 20 linked nucleosides, wherein the modified oligonucleotide has the nucleobase sequence of 5′-GCATCCATTTAATAGAAGTT-3′ (SEQ ID NO:
- 1188), wherein the modified oligonucleotide has a sugar motif of 5′-eeeeeddddddddddeeeee-3′, wherein
- e is a 2′-OCH2CH2OCH3 ribosyl sugar moiety;
- and
- d is a 2′-β-D-deoxyribosyl sugar moiety;
- and
- wherein the modified oligonucleotide has an internucleoside linkage motif of 5′-soooossssssssssooss-3′, wherein
- s is a phosphorothioate internucleoside linkage;
- and
- o is a phosphodiester internucleoside linkage;
- and
- wherein each C is a 5-methylcytosine.
Dependencies are inferred from the claim text.