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IFEZUNTIRGÉN INILPARVOVEC
A RECOMBINANT, REPLICATION DEFICIENT ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5) VECTOR ENCODING A MICRORNA (MIRNA) TARGETING THE EXON 1 OF HUMAN HUNTINGTIN PROTEIN (HTT) MRNA (MIHTT). THE EXPRESSION CASSETTE CONSISTS OF A CHIMERIC CHICKEN BETA-ACTIN PROMOTER, THE MIHTT SEQUENCE ENGINEERED IN THE MIR-451 BACKBONE, WITH THE UPSTREAM REGION OF MIR-451 CONTAINING AN INACTIVATED MIR-144 SEQUENCE, AND A HUMAN GROWTH HORMONE POLYADENYLATION SIGNAL, FLANKED BY AAV2 5' AND 3' INVERTED TERMINAL REPEATS (ITRS)