04-01-2018 дата публикации
Номер: US20180002696A1
Принадлежит:
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Sodium channel, voltage-gated, alpha subunit (SCNA), in particular, by targeting natural antisense polynucleotides of Sodium channel, voltage-gated, alpha subunit (SCNA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of SCNA. 1. A method of upregulating a function of and/or the expression of a Sodium channel , voltage-gated , alpha subunit (SCN1A) gene in mammalian cells or tissues in vivo or in vitro comprising: contacting said cells or tissues with at least one short interfering RNA (siRNA) oligonucleotide 19 to 30 nucleotides in length , said at least one siRNA oligonucleotide being specific for a natural antisense polynucleotide of a Sodium channel , voltage-gated , alpha subunit (SCN1A) polynucleotide of SEQ ID NO: 12 or of SEQ ID NO: 13 , or of SEQ ID NO: 14 , or of SEQ ID NO:15 , or of SEQ ID NO: 16 , or of SEQ ID NO: 17 , or of SEQ ID NO: 18 , or of SEQ ID NO: 19 , or of SEQ ID NO: 20 , or of SEQ ID NO: 21 , or of SEQ ID NO: 22 , or of SEQ ID NO: 23 , or of SEQ ID NO: 24 , or of SEQ ID NO: 25 , or of SEQ ID NO: 26 , or of SEQ ID NO: 27 or of SEQ ID NO: 28; thereby upregulating a function of and/or the expression of the Sodium channel , voltage-gated , type I , alpha subunit (SCN1A) polynucleotide wherein the at least one oligonucleotide comprises at least one oligonucleotide sequences set forth as SEQ ID NOS: 31 , 33 , 37 , 40 to 92.2. A synthetic , modified oligonucleotide of 10 to 30 nucleotides in length comprising at least one modification wherein the at least one modification is selected from: at least one modified sugar moiety; at least one modified internucleotide linkage; at least one modified nucleotide , and combinations thereof; wherein said oligonucleotide is an antisense compound which specifically hybridizes to a ...
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