| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2e3830f35387a1c32061dea3ee310851 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K2319-60
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2800-80
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14145
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-11
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-907
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-22
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-6472 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-864
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N9-22
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-63 |
| filingDate |
2021-04-27-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_e9a2f614155db4d9fc89b729fc0b9751
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f0265e872b156982675234b48faf9926 |
| publicationDate |
2021-11-04-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2021222232-A1 |
| titleOfInvention |
Compositions and uses thereof for treatment of angelman syndrome |
| abstract |
A composition comprising an expression cassette having a nucleic acid sequence encoding one or more elements of a gene editing system that targets UBE3A-ATS on a paternal allele in a neuron of a patient having Angelman syndrome is provided. Also provided is a method for treating one or more symptoms of Angelman syndrome (AS) in a patient having deficient UBE3A expression in neurons, wherein the method comprises delivering a nucleic acid sequence that encodes one or more elements of a gene editing system that targets UBE3A-ATS to modify the UBE3A-ATS coding sequence and provide for expression of paternal UBE3A. |
| priorityDate |
2020-04-28-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |