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DTX-401
7LP92X4WPE
PARIGLASGEN BRECAPARVOVEC
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 8 VECTOR DTX401 HUMAN GLUCOSE 6-PHOSPHATASE ALPHA CODON-OPTIMIZED GENE G6PC PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS)
A NON-REPLICATING ADENO-ASSOCIATED VIRAL VECTOR ENCODING CODON-OPTIMIZED HUMAN GLUCOSE-6-PHOSPHATASE (G6PC). A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 (RAAV8), ENCODING CODON-OPTIMIZED HUMAN GLUCOSE-6-PHOSPHATASE (G6PC) UNDER THE CONTROL OF THE NATIVE HUMAN G6PC PROMOTER/ENHANCER AND AN SV40 POLYADENYLATION SEQUENCE, CONTAINING A CHIMERIC INTRON IN THE 5' UTR BETWEEN THE PROMOTER/ENHANCER AND THE TRANSGENE, FLANKED BY AAV2 INVERTED TERMINAL REPEATS (ITR); REC. LIST 85