C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/713 (2006.01) A61K 48/00 (2006.01) C07H 21/00 (2006.01)
Patent
CA 2684724
The present invention relates to splice switching oligonucleotides or splice switching oligomers (SSOs). The preferred SSOs according to the invention target exon 7 of TNFRl (TNFRSFlA) or TNFR2 (TNFRSFlA) pre-mRNA, typically resulting in the production of TNFR variants which comprise a deletion in part or the entire exon 7 respectfully. SSOs targeting exon 7 are found to result in a soluble form of the TNFR, which has thereputic benefit for treatment of inflammatory diseases. The SSO's are characterized in that they are substantially incapable or incapable of recruiting RNaseH.
La présente invention concerne des oligonucléotides permutant l'épissage ou des oligomères permutant l'épissage (SSO). Les SSO préférés selon l'invention visent l'exon 7 du récepteur TNFR1 (TNFRSF1A) ou TNFR2 (TNFRSF1A) pré-ARNm, entraînant généralement la production de variants du TNFR comprenant une délétion au niveau d'une partie ou de l'ensemble de l'exon 7, respectivement. Les exons 7 visant les SSO permettent d'obtenir une forme soluble du TNFR qui a un effet positif dans le traitement de maladies inflammatoires. Les SSO se caractérisent par le fait qu'ils sont sensiblement incapables ou incapables de recruter la RNaseH.
Oerum Henrik
Sazani Peter L.
Ercole Biotech Inc.
Santaris Pharma A/s
Smart & Biggar
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