| 异名/同义词 | DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TSHA-101 SYNTHETIC CAG PROMOTER PLUS HUMAN .BETA.-N-ACETYLHEXOSAMINIDASE SUBUNIT .BETA. PRECURSOR FUSION PROTEIN WITH PORCINE TESCHOVIRUS 2A PEPTIDE FUSION PROTEIN WITH HUMAN .BETA.-N-ACETYLHEXOSAMINIDASE SUBUNIT .ALPHA. PRECURSOR TRANSGENE PLUS SIMIAN VIRUS 40 POLYADENYLATION SIGNAL PLUS ADENO-ASSOCIATED VIRUS 9 INVERTED TERMINAL REPEAT FLANKS CODON OPTIMIZED-SPECIFYING)
ENGABEXAGENE CINCESPARVOVEC
ENGABEXAGENE CINCESPARVOVEC [INN]
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (RAAV9) VECTOR, ENCODING CODON-OPTIMISED HUMAN .BETA.-HEXOSAMINIDASE SUBUNITS .BETA. AND .ALPHA., JOINED BY A PORCINE TESCHOVIRUS 2A PEPTIDE LINKER, UNDER CONTROL OF A SYNTHETIC PROMOTER (CYTOMEGALOVIRUS (CMV) ENHANCER, CHICKEN .BETA.-ACTIN PROMOTER AND .BETA.-ACTIN INTRON) AND TERMINATED WITH THE SIMIAN VIRUS 40 (SV40) POLYADENYLATION (POLYA) SIGNAL, FLANKED BY ADENO-ASSOCIATED VIRUS SEROTYPE 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS)
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