http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-114466682-A
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_4f733449575d135440f9b3aae7cb4f23 |
| classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 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 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00 |
| 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_0609d3b98a6dda2b190e4b2fc09bb989 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_77d4ed43209f7ce2fcccb08cf48a0b7d |
| publicationDate | 2022-05-10-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | CN-114466682-A |
| titleOfInvention | Nucleic acids targeting exon 44 and recombinant adeno-associated viruses comprising the same for the treatment of dystrophin-based myopathy |
| abstract | The present disclosure relates to the field of gene therapy for the treatment of muscular dystrophies, including but not limited to Duchenne muscular dystrophy (DMD). More specifically, the present disclosure provides nucleic acids, including nucleic acids encoding U7-based small ribonucleic acid (RNA) (snRNA) (U7-based snRNAs), and recombinant adeno-associated viruses (rAAVs) comprising the nucleic acids Molecules to deliver nucleic acids encoding U7-based snRNAs to induce exon skipping for use in the treatment of muscular dystrophy (including but Without limitation DMD), such mutations include, but are not limited to, any mutation involving, surrounding or affecting exon 44 of DMD. |
| 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: 182.