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NEFONADOGÉN VONZUPARVOVEC
NÉFONADOGENE VONZUPARVOVEC
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 2 (RAAV2) VECTOR, ENCODING A MITOCHONDRIAL TARGETING SEQUENCE FROM THE HUMAN HEME A FARNESYLTRANSFERASE GENE (COX10) FUSED TO CODONOPTIMISED HUMAN MITOCHONDRIAL NADH DEHYDROGENASE SUBUNIT 1 (MT-ND1, NADH1, ND1; ALSO KNOWN AS NADHUBIQUINONE OXIDOREDUCTASE CHAIN 1), UNDER CONTROL OF THE CYTOMEGALOVIRUS (CMV) IMMEDIATE EARLY ENHANCER/PROMOTER AND THE SIMIAN VIRUS 40 (SV40) POLYADENYLATION SIGNAL. THE 5' UNTRANSLATED REGION (UTR) ALSO CONTAINS A MACACINE HERPESVIRUS 3 CHIMERIC INTRON, 3' OF THE CMV ENHANCER/PROMOTER AND THE KOZAK SEQUENCE, AND THE 3' UNTRANSLATED REGION COMPRISES THE COX10 3' UTR. THE EXPRESSION CASSETTE IS FLANKED BY AAV2 INVERTED TERMINAL REPEATS