Abstract
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 SSO obtained by said method and uses thereof.
Original language | English |
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IPC | A61K31/7088, C12N15/11,C12N15/113 |
Patent number | AU2022213196 (A1) |
Country/Territory | Denmark |
Priority date | 26/01/2021 |
Priority number | WO2022EP51790 |
Publication status | Published - 10. Aug 2023 |