http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2014143884-A3
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_61f88efcda59d7062a4252ecf6886f58 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-52 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0066 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y504-99002 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-90 |
| classificationIPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
| filingDate | 2014-03-14-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b0080c1b240f16fdb37e7cb865e12d30 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_1795993d833d1d48139bb295800bc4e2 |
| publicationDate | 2014-11-06-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | WO-2014143884-A3 |
| titleOfInvention | Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma) |
| abstract | Synthetic polynucleotides encoding human methylmalonyl-CoA mutase ( synMUT ) and exhibiting augmented expression in cell culture and/or in a subject are described herein. An adeno-associated viral (AAV) gene therapy vector encoding synMUT under the control of a liver-specific promoter (AAV2/8-HCR-hAAT- synMUT -RBG) successfully rescued the neonatal lethal phenotype displayed by methylmalonyl-CoA mutase-deficient mice, lowered circulating methylmalonic acid levels in the treated animals, and resulted in prolonged hepatic expression of the product of synMUT transgene in vivo, human methylmalonyl-CoA mutase (MUT). |
| priorityDate | 2013-03-15-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 43.