http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-4007633-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a6748c1fba1292abba86450fa886727c |
| classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-11 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-32 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-33 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P21-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
| filingDate | 2020-08-03-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_df3214253a32ee5b70572c979d1e679d http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_e421bd94793e16e1ab135bae9b6c9cdb |
| publicationDate | 2022-06-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | EP-4007633-A1 |
| titleOfInvention | Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies |
| abstract | The disclosure relates to the field of gene therapy for the treatment of a muscular dystrophy including, but not limited to, Duchenne Muscular Dystrophy (DMD). More particularly, the disclosure provides nucleic acids, including nucleic acids encoding U7-based small nuclear ribonucleic acids (RNAs) (snRNAs), U7-based snRNAs, and recombinant adeno-associated virus (rAAV) comprising the nucleic acid molecules to deliver nucleic acids encoding U7-based snRNAs to induce exon-skipping for use in treating a muscular dystrophy including, but not limited to, DMD, resulting from a mutation amenable to skipping exon 44 of the DMD gene (DMD exon 44) including, but not limited to, any mutation involving, surrounding, or affecting DMD exon 44. |
| priorityDate | 2019-08-02-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 201.