| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2a819eda0adf22936a52362eeebb9fb4 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2227-105
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K47-26
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2800-22
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2267-0306
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2840-007
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-42
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-50
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0019
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2217-075 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-50
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y305-03001
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K67-0275
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-7088
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-28
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0058 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-28
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-7088 |
| filingDate |
2020-05-07-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f4bdc5f7e57634e42f357c68a58a7f47 |
| publicationDate |
2020-11-12-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2020227542-A1 |
| titleOfInvention |
A method to prevent the myelin abnormalites associated with arginase deficiency |
| abstract |
The invention disclosed herein provides methods and materials useful in gene therapy regimens designed to inhibit myelination abnormalities that occur in the urea cycle disorder arginase deficiency. The underlying cause of the progressive neurological dysfunction that occurs in this disorder has been previously unknown and conventional therapies, at best, only slow the onset of neurological dysfunction. This neurological dysfunction results at least in part from the dysmyelination that occurs in the central nervous system due to the lack of adequate hepatic expression of arginase 1. We have discovered an origin of this neurological dysfunction and, using this information, designed materials and associated methods of gene therapy. The methods and materials disclosed herein can inhibit and essentially prevent neurological dysfunction in a murine model of arginase deficiency. |
| priorityDate |
2019-05-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |