| 异名/同义词 | DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TSHA-118 CHICKEN .BETA.-ACTIN HYBRID PROMOTER PLUS HUMAN ACYL PROTEIN THIOESTER HYDROLASE PPT1 PRECURSOR TRANSGENE PLUS BOVINE GROWTH HORMONE POLYADENYLATION SIGNAL PLUS ADENO-ASSOCIATED VIRUS 9 INVERTED TERMINAL REPEAT FLANKS-SPECIFYING)
RECOMBINANT, SELF-COMPLEMENTARY (DIMERIC), NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (SCAAV9) VECTOR, ENCODING CODON-OPTIMISED HUMAN PALMITOYL-PROTEIN THIOESTERASE 1 (PPT1, CLN1), UNDER THE CONTROL OF A SYNTHETIC PROMOTER CONSISTING OF ACYTOMEGALOVIRUS (CMV) ENHANCER/CHICKEN .BETA.-ACTIN (CBA) PROMOTER AND A HYBRID INTRON DERIVED FROM CHICKEN .BETA.-ACTIN AND MINUTE VIRUS OF MICE (MVM) INTRONS, FOLLOWED BY A KOZAK SEQUENCE, AND TERMINATED BY A BOVINE GROWTH HORMONE (BGH) POLYADENYLATION (POLYA) SIGNAL AND THE VECTOR GENOME CONTAINS A MUTATED ADENO-ASSOCIATED VIRUS SEROTYPE 2 (AAV2) INVERTED TERMINAL REPEAT (MITR) AND IS FLANKED BY AAV2 WILD-TYPE INVERTED TERMINAL REPEATS (WTITRS)
ZAFTUCLENEGENE PIRUPARVOVEC
ZAFTUCLENEGENE PIRUPARVOVEC [INN]
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