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ENGABEXAGÉN CINCESPARVOVEC
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 ADENOASSOCIATED VIRUS SEROTYPE 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS)