(INNS)
BIDRIDISTROGEN XEBOPARVOVEK(INNC)
RECOMBINANT SELF-COMPLEMENTARY (DIMERIC) NON-REPLICATING ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE RH.74 VECTOR ENCODING CODON-OPTIMIZED HUMAN BETA-SARCOGLYCAN (HSGCB) UNDER CONTROL OF A HYBRID PROMOTER (MHCK7) CONSISTING OF ENHANCER/PROMOTER REGIONS OF MURINE MUSCLE CREATINE KINASE (CK) AND ALPHA-MYOSIN HEAVYCHAIN GENES, FOLLOWED BY A CHIMERIC INTRON, COMPOSED OF THE 5' DONOR SITE FROM THE HUMAN BETA-GLOBIN GENE AND THE BRANCHPOINT AND 3' SPLICE ACCEPTOR SITE FROM IGG HEAVY CHAIN VARIABLE REGION, AND TERMINATED BY A SMALL SYNTHETIC POLYADENYLATION (POLYA) SIGNAL SEQUENCE, FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS)(WHO)
BIDRIDISTROGENUM XEBOPARVOVECUM(INN)