| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_6e09319fe979c0d63f50e0b9dcf132ae |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-11
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-33 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0058
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0066
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-62
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P21-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-62 |
| filingDate |
2016-12-09-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| grantDate |
2020-08-25-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_a5eb1adba5f6890046932071a79bc894
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b617860e230f1958a4842643298e7c3a |
| publicationDate |
2020-08-25-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
US-10752898-B2 |
| titleOfInvention |
Effective gene therapy tools for dystrophin exon 53 skipping |
| abstract |
The invention relates to a recombinant adeno-associated viral vector (rAAV) comprising a sequence encoding an antisense oligonucleotide (AON) directed against a segment of at least 33 bases from the +30 to +69 region of exon 53 of the pre-messenger RNA (pre-mRNA) of dystrophin, advantageously of human origin, and to the use thereof as a drug, in particular for the treatment of Duchenne muscular dystrophy (DMD). |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11787869-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11679161-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11168141-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11248056-B1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11369689-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11633496-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11497815-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11771776-B2 |
| priorityDate |
2015-12-09-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |