SPLICE SWITCHING OLIGONUCLEOTIDES TARGETING PSEUDOEXONS

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'...

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Bibliographic Details
Main Authors PETERSEN, Ulrika Simone Spangsberg, HOLM, Lise, Lolle, ANDRESEN, Brage, Storstein, BRUUN, Gitte, Hoffmann, DOKTOR, Thomas, Koed
Format Patent
LanguageEnglish
French
German
Published 06.12.2023
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Summary: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.
Bibliography:Application Number: EP20220702677