Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a6748c1fba1292abba86450fa886727c |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-008 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4716 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 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/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07K14-47 |
filingDate |
2020-08-21-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_dba6fe32ed46ec4283a0bd81cfe2d700 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b2761ec2c1bacb3e67b536bc1ee711a7 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ae5411943a45c0f81af675535608383b |
publicationDate |
2021-02-25-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
WO-2021035120-A1 |
titleOfInvention |
Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
abstract |
Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.hSCGA using a systemic route of administration and at a dose of about 1.0 x 1012 vg/kg to about 5.0 x 1015 vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-4198046-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2023111102-A1 |
priorityDate |
2019-08-21-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |