| 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/A61K38-47
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-861
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-2402
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0075 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N9-24
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86 |
| filingDate |
2020-10-22-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_6982d05c7d915fd5c6d87044a19e04da
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f0265e872b156982675234b48faf9926 |
| publicationDate |
2021-04-29-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2021081217-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. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021231579-A1 |
| priorityDate |
2019-10-23-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |