FDA全球物质注册数据库(FDA Global Substance Registration System)
物质名称
RETOFRAXGENE PARVEC
异名/同义词
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE RHESUS 10 (RAAVRH.10) VECTOR ENCODING THE FULL-LENGTH HUMAN FRATAXIN (FXN) PRECURSOR PROTEIN PRECEDED BY A KOZAK SEQUENCE. TRANSGENE EXPRESSION IS UNDER CONTROL OF THE SYNTHETIC CAG PROMOTER (HUMAN CYTOMEGALOVIRUS ENHANCER, CHICKEN .BETA.-ACTIN PROMOTER, RABBIT .BETA.-GLOBIN INTRON) AND TERMINATED WITH A RABBIT .BETA.-GLOBIN POLYADENYLATION SIGNAL; FLANKED BY AAV2 INVERTED TERMINAL REPEATS (ITRS) RETOFRAXGENE PARVEC RETOFRAXGENE PARVEC [INN]