| Predicate |
Object |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-33
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3235
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-11
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3231
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-315 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-111
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12Q1-68
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P21-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12Q1-02
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-711
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
| filingDate |
2012-11-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationDate |
2014-12-18-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
JP-2014533944-A |
| titleOfInvention |
Compounds for modulation of SMN2 splicing |
| abstract |
The present invention relates to oligomeric compounds (oligomers) that target human SMN2 encoding nucleic acids in cells and effect modulation of SMN2 mRNA splicing that favors full-length SMN2 mRNA but not the fully functional truncated transcript SMN2Δ7 . Decreased SMNΔ7 mRNA expression and / or increased full-length SMN2 mRNA expression is beneficial for the treatment of diseases or disorders associated with abnormal forms of SMN2, particularly overexpression or undesirable high levels of SMN2Δ7, such as spinal muscular atrophy (SMA) It is. [Selection figure] None |
| priorityDate |
2011-11-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |