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Compounds and methods for reducing kcnt1 expression
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A technology of oligomeric compounds and substitutes, applied in biochemical equipment and methods, drug combinations, recombinant DNA technology, etc., can solve problems such as increased peak potassium current and increased potassium channel activity
Pending Publication Date: 2021-11-16
IONIS PHARMA INC
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These missense mutations lead to increased potassium channel activity and peak potassium current
Method used
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Embodiment approach 1
[0071] Embodiment 1. A kind of oligomeric compound, described oligomeric compound comprises the oligonucleotide of the modification that is made up of the nucleoside of 12 to 50 connections, wherein the nucleobase sequence of the oligonucleotide of the modification and KCNT1 The isometric portion of the nucleic acid is at least 90% complementary, and wherein said modified oligonucleotide comprises at least one modification selected from a modified sugarmoiety and a modified internucleoside linkage.
Embodiment approach 2
[0072] Embodiment 2. An oligomeric compound comprising a modified oligonucleotide consisting of 12 to 50 linked nucleosides and having a compound comprising SEQ ID NO: 21- A nucleobase sequence of at least 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19 or 20 contiguous nucleobases of any of 2939.
Embodiment approach 3
[0073] Embodiment 3. An oligomeric compound comprising a modified oligonucleotide consisting of 12 to 50 linked nucleosides and comprising at least 8, at least 9 at least 10, at least 11, at least 12, at least 13, at least 14, at least 15, at least 16, at least 17, at least 18, at least 19 or at least 20 contiguous nucleobases nucleobase sequence, these contiguous nucleobases are complementary to:
[0074] an isometric portion of nucleobases 24523-24561 of SEQ ID NO:2,
[0075] an isometric portion of nucleobases 27568-27603 of SEQ ID NO:2,
[0076] an isometric portion of nucleobases 30772-30811 of SEQ ID NO:2,
[0077] an isometric portion of nucleobases 54372-54428 of SEQ ID NO:2,
[0078] an isometric portion of nucleobases 55785-55818 of SEQ ID NO:2,
[0079] an isometric portion of nucleobases 56048-56073 of SEQ ID NO:2,
[0080] an isometric portion of nucleobases 56319-56349 of SEQ ID NO:2,
[0081] an isometric portion of nucleobases 57683-57710 of SEQ ID NO:2, ...
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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 lobeepilepsy (ADNFLE), West syndrome, and Ohtahara syndrome.
Description
[0001] sequence listing [0002] This application is filed with a sequence listing in electronic format. The sequence listing is provided as a file titled BIOL0358WOSEQ_ST25.txt created on March 9, 2020 and is 716 kb in size. The entire content of the information in the electronic format of the Sequence Listing is incorporated herein by reference. technical field [0003] Compounds, methods, and medicaments for reducing the amount of sodium-activated potassium channel subfamily T member 1 (KCNT1 ) RNA in a cell or a subject, and in some cases reducing the amount of KCNT1 protein in a cell or a subject are provided combination. These compounds, methods and pharmaceutical compositions are useful for ameliorating at least one symptom or marker of a neurological disorder. These symptoms and signs include (but are not limited to) encephalopathy, cerebral cortical atrophy, clonus, seizures (epilepsy), and behavioral abnormalities such as aggression, catatonia, psychosis, and othe...
Claims
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