Allele specific splice switching oligonucleotides targeting pseudoexons

Brage Storstein Andresen; Thomas Koed Doktor; Lise Lolle Holm; Ulrika Simone Spangsberg Petersen; Gitte Hoffmann Bruun

Allele specific splice switching oligonucleotides targeting pseudoexons

Brage Storstein Andresen (Inventor), Thomas Koed Doktor (Inventor), Lise Lolle Holm (Inventor), Ulrika Simone Spangsberg Petersen (Inventor), Gitte Hoffmann Bruun (Inventor)

Research output: 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).

Original language	English
IPC	A61K31/7088,C12N15/11,C12N15/113
Patent number	WO2023144263 (A3)
Country/Territory	Denmark
Priority date	26/01/2022
Priority number	DK2022PA70346
Publication status	Published - 3. Aug 2023

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title = "Allele specific splice switching oligonucleotides targeting pseudoexons",

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).",

author = "Andresen, {Brage Storstein} and Doktor, {Thomas Koed} and Holm, {Lise Lolle} and Petersen, {Ulrika Simone Spangsberg} and Bruun, {Gitte Hoffmann}",

year = "2023",

month = aug,

day = "3",

language = "English",

type = "Patent",

note = "WO2023144263 (A3); A61K31/7088,C12N15/11,C12N15/113",

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TY - PAT

T1 - Allele specific splice switching oligonucleotides targeting pseudoexons

AU - Andresen, Brage Storstein

AU - Doktor, Thomas Koed

AU - Holm, Lise Lolle

AU - Petersen, Ulrika Simone Spangsberg

AU - Bruun, Gitte Hoffmann

PY - 2023/8/3

Y1 - 2023/8/3

N2 - 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).

AB - 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).

M3 - Patent

M1 - WO2023144263 (A3)

ER -

Allele specific splice switching oligonucleotides targeting pseudoexons

Abstract

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