| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_7a9da612c0ac08c248dec5bc3bfeff3a |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14152
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2227-105
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2710-14052
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-531
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-30
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-31
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-141
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2267-0306
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2710-14045
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14122
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K2319-10 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N7-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0075
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-015
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-47
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-04
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-005
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/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P21-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86 |
| filingDate |
2020-02-28-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_dde2cdf275be1e8a3a3c74058749cb94
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_9a31c385d7b8dd119a6cb5a61766f24b
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_2cf5a72e145d368430fb4c3909a2c7fa
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d41fd82bdc01a1d06629e47e8ce3ff74 |
| publicationDate |
2022-03-31-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
US-2022098614-A1 |
| titleOfInvention |
Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) |
| abstract |
The present disclosure relates to modified adeno-associated virus (AAV) delivery vectors comprising ‘silence and replace’ DNA constructs, compositions comprising same, and the use of the modified AAV and compositions to treat oculopharyngeal muscular dystrophy (OPMD) in individuals suffering from OPMD or which are predisposed thereto. In particular, the disclosure relates to AAV having a capsid protein with a modified subunit 1 (VP1) and comprising a ‘silence and replace’ DNA construct, wherein the ‘silence and replace’ DNA construct comprises (i) a DNA-directed RNAi (ddRNAi) construct encoding short hairpin microRNA (shmiR) targeting PABPN1 causative of OPMD and (ii) a PABPN1 construct encoding functional PABPN1 protein having a mRNA transcript which is not targeted by the shmiRs at (i). The present disclosure also relates to the methods of treating OPMD comprising direct injection of an AAV of the disclosure to a subject's pharyngeal muscles. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2022106594-A1 |
| priorityDate |
2019-02-28-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |