Allele specific splice switching oligonucleotides targeting pseudoexons

Publikation: Patent

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 SSOs obtained by said method and uses thereof. In particular, the present invention relates to allele specific splice switching oligonucleotides (SSOs).

OriginalsprogEngelsk
IPCA61K31/7088,C12N15/11,C12N15/113
PatentnummerWO2023144263 (A3)
Land/OmrådeDanmark
Prioritetsdato26/01/2022
PrioritetsnummerDK2022PA70346
StatusUdgivet - 3. aug. 2023

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