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DOMOFENOGÉN ZALFAPARVOVEC
DOMOFÉNOGENE ZALFAPARVOVEC
RECOMBINANT, REPLICATION-INCOMPETENT ADENOASSOCIATED VIRUS SEROTYPE 5 (AAV5) VECTOR ENCODING CODON-OPTIMIZED HUMAN PHENYLALANINE HYDROXYLASE (HPAH) UNDER CONTROL OF A LIVER-SELECTIVE TRANSCRIPTION REGULATORY ELEMENT (TRE), CONSISTING OF THE APOLIPOPROTEIN E ENHANCER AND THE HUMAN ALPHA-1 ANTI-TRYPSIN PROMOTER, A HUMAN ALPHA-1 ANTI-TRYPSIN/HUMAN BETA-GLOBIN HYBRID INTRON AND TERMINATED BY THE BOVINE GROWTH HORMONE POLYADENYLATION SEQUENCE, FLANKED BY AAV2 INVERTED TERMINAL REPEATS (ITRS)