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'...
Saved in:
Main Authors | , , , , |
---|---|
Format | Patent |
Language | English |
Published |
10.08.2023
|
Subjects | |
Online Access | Get full text |
Cover
Loading…
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: AU20220213196 |