ALLELE SPECIFIC 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 French |
Published |
03.08.2023
|
Subjects | |
Online Access | Get full text |
Cover
Loading…
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).
La présente invention concerne un procédé d'identification d'oligonucléotides à commutation d'épissage (SSO) pouvant moduler l'expression d'une protéine cible dans une cellule en favorisant l'incorporation d'un pseudoexon dans l'ARNm mature lors de la liaison au pré-ARNm dans une région située entre +9 et +39 en aval du site d'épissage 5' dudit pseudoexon. L'invention concerne également les SSO obtenus par ledit procédé et ses utilisations. En particulier, la présente invention concerne des oligonucléotides de commutation d'épissage (SS) spécifiques d'allèles. |
---|---|
AbstractList | 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).
La présente invention concerne un procédé d'identification d'oligonucléotides à commutation d'épissage (SSO) pouvant moduler l'expression d'une protéine cible dans une cellule en favorisant l'incorporation d'un pseudoexon dans l'ARNm mature lors de la liaison au pré-ARNm dans une région située entre +9 et +39 en aval du site d'épissage 5' dudit pseudoexon. L'invention concerne également les SSO obtenus par ledit procédé et ses utilisations. En particulier, la présente invention concerne des oligonucléotides de commutation d'épissage (SS) spécifiques d'allèles. |
Author | BRUUN, Gitte Hoffmann ANDRESEN, Brage Storstein PETERSEN, Ulrika Simone Spangsberg HOLM, Lise Lolle DOKTOR, Thomas Koed |
Author_xml | – fullname: PETERSEN, Ulrika Simone Spangsberg – fullname: BRUUN, Gitte Hoffmann – fullname: HOLM, Lise Lolle – fullname: DOKTOR, Thomas Koed – fullname: ANDRESEN, Brage Storstein |
BookMark | eNrjYmDJy89L5WRwd_TxcfVxVQgOcHX2dPN0BjJ8PJ2B_HDPEGcPTz93BX8fT3d_v1BnH1f_EE8X12CFEMcgd9cQkFRAsGuoi79rhL9fMA8Da1piTnEqL5TmZlB2cwWaoJtakB-fWlyQmJyal1oSH-5vZGBkbGhiYmRm7GhoTJwqAOTFL7I |
ContentType | Patent |
DBID | EVB |
DatabaseName | esp@cenet |
DatabaseTitleList | |
Database_xml | – sequence: 1 dbid: EVB name: esp@cenet url: http://worldwide.espacenet.com/singleLineSearch?locale=en_EP sourceTypes: Open Access Repository |
DeliveryMethod | fulltext_linktorsrc |
Discipline | Medicine Chemistry Sciences |
DocumentTitleAlternate | OLIGONUCLÉOTIDES À COMMUTATION D'ÉPISSAGE SPÉCIFIQUES D'ALLÈLE CIBLANT DES PSEUDOEXONS |
ExternalDocumentID | WO2023144263A1 |
GroupedDBID | EVB |
ID | FETCH-epo_espacenet_WO2023144263A13 |
IEDL.DBID | EVB |
IngestDate | Fri Aug 09 05:00:55 EDT 2024 |
IsOpenAccess | true |
IsPeerReviewed | false |
IsScholarly | false |
Language | English French |
LinkModel | DirectLink |
MergedId | FETCHMERGED-epo_espacenet_WO2023144263A13 |
Notes | Application Number: WO2023EP51916 |
OpenAccessLink | https://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20230803&DB=EPODOC&CC=WO&NR=2023144263A1 |
ParticipantIDs | epo_espacenet_WO2023144263A1 |
PublicationCentury | 2000 |
PublicationDate | 20230803 |
PublicationDateYYYYMMDD | 2023-08-03 |
PublicationDate_xml | – month: 08 year: 2023 text: 20230803 day: 03 |
PublicationDecade | 2020 |
PublicationYear | 2023 |
RelatedCompanies | SYDDANSK UNIVERSITET |
RelatedCompanies_xml | – name: SYDDANSK UNIVERSITET |
Score | 3.4900765 |
Snippet | 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... |
SourceID | epo |
SourceType | Open Access Repository |
SubjectTerms | 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 |
Title | ALLELE SPECIFIC SPLICE SWITCHING OLIGONUCLEOTIDES TARGETING PSEUDOEXONS |
URI | https://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20230803&DB=EPODOC&locale=&CC=WO&NR=2023144263A1 |
hasFullText | 1 |
inHoldings | 1 |
isFullTextHit | |
isPrint | |
link | http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwfV3dS8MwED_GFPVNp-J0SkHpW7Fdu5k-DOnSdFa6pqyd69uwHwNBuuEq_vtewqZ72lsuR45wcPndLx8XgIduUSBIFUTLEU80q08Wmk0MU0PqbOs5KUxDFvUZh_2XqfWa9tIGfG7fwsg6oT-yOCJGVI7xXsv1evW_ieXKu5Xrx-wDu5bPXjJw1Q07xnya6KbqDgcs4i6nKqXI29RwInXIHbp900GudCASaVFpn70NxbuU1S6oeKdwGKG9qj6DRlm14Jhu_15rwdF4c-SNzU30rc9h5AQBC5gSR4z6nk-xEfgU5ZmfULHtpPDAH_FwSgPGE99lsZI4kxFLhCqK2dTlLOVhfAH3HsMRGs5o_ueA-YzvTt-8hGa1rMorUJ4IJih6r9Az27YWiL2Cb5ZWSd5tI0Nvt6Gzz9L1fvUNnAhRXnczO9Csv77LW4TgOruTnvsFczyB0Q |
link.rule.ids | 230,309,786,891,25594,76903 |
linkProvider | European Patent Office |
linkToHtml | http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwfV3fT8IwEL4QNOKbosYfqEs0e1scbED3QAx0HVTLurAhvBHHRmJiBpEZ_31vDShPvLW99NJccv36Xa9XgMdGkiBIJcSYI54YdossDIfULQOps2POSWLVVVGfod8ajO2XaXNags_tWxhVJ_RHFUdEj5qjv-dqv179B7FclVu5foo_cGj57EUdV9-wYzxPE9PS3V6HBdKVVKcUeZvuj5QMuUOjZXWRKx20kRQqsvTWK96lrHZBxTuBwwD1ZfkplNKsChW6_XutCkfDzZU3Njfetz6DflcIJpgWBoxyj1NsCE6xP-ERLcJOmhS8L_0xFUxG3GWhFnVHfRYVoiBkY1eyqfTDc3jwGM4wcEWzPwPMJnJ3-dYFlLNlll6C1iZ4QDGbiRk7jr1A7C34Zmqn5N2px2jtK6jt03S9X3wPlUE0FDPB_dcbOC5EKvXNqkE5__pObxGO8_hOWfEXqiaEuw |
openUrl | ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Apatent&rft.title=ALLELE+SPECIFIC+SPLICE+SWITCHING+OLIGONUCLEOTIDES+TARGETING+PSEUDOEXONS&rft.inventor=PETERSEN%2C+Ulrika+Simone+Spangsberg&rft.inventor=BRUUN%2C+Gitte+Hoffmann&rft.inventor=HOLM%2C+Lise+Lolle&rft.inventor=DOKTOR%2C+Thomas+Koed&rft.inventor=ANDRESEN%2C+Brage+Storstein&rft.date=2023-08-03&rft.externalDBID=A1&rft.externalDocID=WO2023144263A1 |