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PATIDISTROGÉN BEXOPARVOVEC
RECOMBINANT SELF-COMPLEMENTARY (DIMERIC) NONREPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE RH.74 (AAVRH74) VECTOR ENCODING CODON-OPTIMIZED HUMAN ALPHA-SARCOGLYCAN (HSGCA), UNDER THE CONTROL OF A TRIPLE E BOX MUSCLE CREATINE KINASE (TMCK) PROMOTER/ENHANCER, FOLLOWED BY A CHIMERIC INTRON,) COMPOSED OF THE 5' DONOR SITE FROM THE HUMAN BETAGLOBIN GENE AND THE BRANCHPOINT AND 3' SPLICE ACCEPTOR SITE FROM THE 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)