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filingDate 2022-04-12-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ba14904114a21b3990d5ea2026ca065e
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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.
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http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID396442
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP24855
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID450133
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02126
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID8467
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP14392
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02125
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID451367506
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP14391
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02130
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ6QM28
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP14524
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID23670522
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02129
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP14521
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID419491714
http://rdf.ncbi.nlm.nih.gov/pubchem/anatomy/ANATOMYID9986
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02134
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID403580
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP09907
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP75358
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02131
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID403588
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID451761003
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP14526
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP09840
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID2783

Total number of triples: 666.