| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2e3830f35387a1c32061dea3ee310851 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y302-01076
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y302-01076
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0075
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-2402
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-47
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-861 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-861
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N9-24 |
| filingDate |
2020-10-22-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_09fc4ac0c4e08cf51f4d4e410ec498d2
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_6982d05c7d915fd5c6d87044a19e04da |
| publicationDate |
2022-12-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
US-2022389457-A1 |
| titleOfInvention |
Compositions for drg-specific reduction of transgene expression |
| abstract |
Provided herein are nucleic acid sequence encoding hIDUA and expression cassettes containing these coding sequences. Also provided are vectors, such as recombinant adeno-associated virus (rAAV) vectors having a vector genome including a hIDUA coding sequence operably linked regulatory sequences that direct expression of the hIDUA. Also provided are compositions containing these expression cassettes and rAAV vectors and methods of treating MPS1 or an associated syndrome such as Hurler, Hurler-Scheie and/or Scheie syndrome. The compositions and methods provided are further designed to selectively repress expression of hIDUA in dorsal root ganglia. |
| priorityDate |
2019-10-23-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |