Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2e3830f35387a1c32061dea3ee310851 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-141 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2330-50 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-1138 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
filingDate |
2022-04-12-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ba14904114a21b3990d5ea2026ca065e http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_41ec3a13f7f8cebd8f1cbb79dc38dda9 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f0265e872b156982675234b48faf9926 |
publicationDate |
2022-10-20-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
WO-2022221276-A1 |
titleOfInvention |
Compositions useful for treating spinal and bulbar muscular atrophy (sbma) |
abstract |
Compositions useful for treatment of Spinal and Bulbar Muscular Atrophy (SBMA) comprising administration of a recombinant adeno-associated virus (rAAV) vector having an AAV capsid and a vector genome comprising a sequence encoding at least one hairpin forming miRNA that comprises a targeting sequence which binds a target site on the mRNA of human androgen receptor, wherein the miRNA inhibits expression of human androgen receptor, is provided. Also provided are compositions containing a rAAV vector and methods of treating SBMA in patient comprising administration of a rAAV vector. |
priorityDate |
2021-04-12-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |