DNA (RECOMBINANT REPLICATION-DEFICIENT ADENO-ASSOCIATED VIRUS 5 VECTOR BMN 307 HUMAN LIVER-SPECIFIC TRANSCRIPTION REGULATORY ELEMENT PLUS HUMAN PHENYLALANINE 4-HYDROXYLASE CODON-OPTIMIZED GENE HPAH PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT F
Common Name
English
domofenogene zalfaparvovec [INN]
Common Name
English
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 APOLIPOPROTEI
