| 异名/同义词 | A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV REP2-CAP6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN IDURONATE 2-SULFATASE (HIDS) TRANSGENE CASSETTE, ENCODING PARTS OF EXON 1 PLUS EXONS 2-9 FROM HIDS AND A SPLICE ACCEPTOR SITE (SA) DERIVED FROM HUMAN COAGULATION FACTOR IX (HF9, FACTOR IX OR FIX) EXON 2, FLANKED BY A SEQUENCE HOMOLOGOUS TO THE ZINC-FINGER NUCLEASE (ZFN) CLEAVAGE SITE OF THE HUMAN ALBUMIN (HALB) INTRON 1
SB-913
TEFIDSOGENE CIVAPARVOVEC
TEFIDSOGENE CIVAPARVOVEC [INN]
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