Allele specific splice switching oligonucleotides targeting pseudoexons

Research output: Patent


The present invention relates to a method for identifying splice switching oligonucleotides (SSOs) able to modulate expression of a target protein in a cell by promoting incorporation of a pseudoexon into the mature mRNA upon binding to the pre-mRNA in the region +9 to +39 downstream to the 5' splice site of said pseudoexon. The invention also relates to SSOs obtained by said method and uses thereof. In particular, the present invention relates to allele specific splice switching oligonucleotides (SSOs).

Original languageEnglish
Patent numberWO2023144263 (A3)
Priority date26/01/2022
Priority numberDK2022PA70346
Publication statusPublished - 3. Aug 2023


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