| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_75694144c0996a751383f3e417644316 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-11
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2330-51
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-32
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-33 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Q1-68
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-8645
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-10
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12Q1-68
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-11
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-09 |
| filingDate |
2017-02-24-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_da3b9e67874f355af294e4a69439d981 |
| publicationDate |
2017-08-31-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2017147467-A1 |
| titleOfInvention |
Recombinant virus products and methods for inducing dux4 exon skipping |
| abstract |
The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nuclear RNAs to induce DUX4 exon- skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022115745-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-114026234-A
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021010301-A1 |
| priorityDate |
2016-02-26-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |