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.
Originalsprog | Engelsk |
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IPC | A61K31/7088, C12N15/11,C12N15/113 |
Patentnummer | AU2022213196 (A1) |
Land/Område | Danmark |
Prioritetsdato | 26/01/2021 |
Prioritetsnummer | WO2022EP51790 |
Status | Udgivet - 10. aug. 2023 |