| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_461514df460e0cdd5644e86ba60adbf7
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_4fd07571555396ab21f333e34fcbf12e
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_42dcb8afd289e295b65f450020cbfdb4 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y401-01028 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-88
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-4515
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-5513
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-5184
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-625
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-8645
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-8509
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0083
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/A61K31-4515
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N9-88
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K9-51
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-864
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-85
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-62
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-5513 |
| filingDate |
2018-04-12-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| grantDate |
2021-01-26-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b3605d7f6f303134f497a5d88088b5d1
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_990ee12ac58ed480f099f2ee138fd90d
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_7315219cd579dd200957ba81ee247c6a |
| publicationDate |
2021-01-26-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
US-10898585-B2 |
| titleOfInvention |
Gene therapy for AADC deficiency |
| abstract |
The present invention is directed to compositions and methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8×1011 vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4×1011 vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7×1011 vg/mL. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the method optionally further comprises the step of administering a therapeutically effective dose of dopamine-antagonist to the subject such as risperidone. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the method optionally comprises providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, and empty capsids. |
| priorityDate |
2017-04-14-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |