Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2e3830f35387a1c32061dea3ee310851 |
classificationCPCAdditional |
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/A61K48-0075 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0058 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0041 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-00 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y204-02008 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-1077 |
classificationIPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-09 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N7-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-63 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
filingDate |
2022-06-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_41ec3a13f7f8cebd8f1cbb79dc38dda9 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ba14904114a21b3990d5ea2026ca065e http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_014d5d603264ae43cb05a047fa4d2a19 |
publicationDate |
2022-12-15-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
WO-2022261187-A1 |
titleOfInvention |
Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof |
abstract |
A rAAV for treatment of a disorder associated with a deficiency in hypoxanthine- guanine phosphoribosyltransferase (HPRT) enzyme levels via gene delivery is provided herein. Also provided herein is a method for treating disorder associated with a deficiency in hypoxanthine-guanine phosphoribosyltransferase (HPRT) enzyme levels by direct delivery to dopaminergic neurons. Correction of Lesch-Nyhan disease may be monitored by assessing dopamine metabolism levels and/or imaging for the presence of dopaminergic cell bodies. |
priorityDate |
2021-06-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |