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DELANDISTROGÉN MOXEPARVOVEC
DÉLANDISTROGENE MOXÉPARVOVEC
A NON-REPLICATING ADENO-ASSOCIATED VIRUS (AAV) VECTOR ENCODING HUMAN MICRO-DYSTROPHIN. A RECOMBINANT, NON-REPLICATING, ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE RH74 VECTOR (AAVRH74) ENCODING CODON-OPTIMIZED HUMAN MICRO-DYSTROPHIN (A SHORTENED FUNCTIONAL VERSION OF HUMAN DYSTROPHIN), UNDER CONTROL OF A HYBRID MHCK7 PROMOTER CONSISTING OF ENHANCER/PROMOTER REGIONS OF MURINE MUSCLE CREATINE KINASE (CK) AND ALPHA-MYOSIN HEAVY-CHAIN GENES, FOLLOWED BY A CHIMERIC INTRON [INCLUDING THE SV40 LATE 16S/19S SPLICE SIGNALS AND A SMALL 5' UTR] AND A SMALL SYNTHETIC POLYADENYLATION (POLYA) SIGNAL SEQUENCE, FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS). PROPOSED INN: LIST 124, REC. INN: LIST 86