| 异名/同义词 | A NON-REPLICATING, RECOMBINANT ADENO-ASSOCIATED VIRUS, HEMATOPOIETIC STEM CELL SEROTYPE 15 (AAVHSC15) EXPRESSING CODON-OPTIMISED HUMAN PHENYLALANINE HYDROXYLASE (PAH) UNDER CONTROL OF A LIVER-SPECIFIC HYBRID PROMOTER (COMPRISING HUMAN HEPATIC CONTROL REGION-1 (HCR-1) ENHANCER ELEMENT, HUMAN ALPHA-1-ANTITRYPSIN (HAAT) PROMOTER AND THE SV40 SMALL T INTRON)) AND AN SV40 POLYA SIGNAL SEQUENCE, FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS)
BEVUFENOGENE NOFEPARVOVEC
BEVUFENOGENE NOFEPARVOVEC [INN]
BEVUFENOGENE NOFEPARVOVEC [USAN]
DNA (RECOMBINANT HUMAN HEMATOPOIETIC STEM CELL-DERIVED ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE AAVHSC15 HUMAN LIVER-SPECIFIC EXPRESSION CASSETTE INCLUDING HUMAN PHENYLALANINE 4-HYDROXYLASE CODON-OPTIMIZED CDNA PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS)
HMI-102
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