Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a034b464ad48f980cfb60072dbd01019 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_82787c2fee17b0b0eb295d5af7d2325f |
classificationIPCInventive |
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/A61K38-45 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P43-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K35-76 |
filingDate |
2019-12-20-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_72e9b6d1be809d9b9c3b3d3fbcd2a8c8 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_fd06f68134731da0a879ca257b9ecc10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_de8caa590b3a2d76c70106a80eab734c http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_21e68fbc04e70b6aaf7d173e69eb25e5 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f69df54ad1f9aac9593b8d4db29c1779 |
publicationDate |
2021-07-01-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
JP-2021097617-A |
titleOfInvention |
Adeno-associated virus virion for the treatment of ornithine transcarbamylase deficiency |
abstract |
PROBLEM TO BE SOLVED: To provide a novel therapeutic means for ornithine transcarbamylase deficiency. The present application is a modified adeno-associated virus vector that expresses ornithine transcarbamylase, reduces attack from neutralizing antibodies in blood, and efficiently transgenes into the liver of a living body (for example, human). I will provide a. The present application comprises a modified VP1 protein that expresses ornithine transcarbamylase and comprises an amino acid sequence in which at least one of the 472nd, 587th and 706th positions in the amino acid sequence of the VP1 protein is replaced with another amino acid. , A modified adeno-associated virus vector that does not cross-react with a neutralizing antibody against AAV2. [Selection diagram] None |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022224372-A1 |
priorityDate |
2019-12-20-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |