(INNS)
ZAFTUKLENEGEN PIRUPARVOVEK(INNC)
RECOMBINANT, SELF-COMPLEMENTARY (DIMERIC), NONREPLICATING 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 A CYTOMEGALOVIRUS (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; THE VECTOR GENOME CONTAINS A MUTATED ADENO-ASSOCIATED VIRUS SEROTYPE 2 (AAV2) INVERTED TERMINAL REPEAT (MITR) AND IS FLANKED BY AAV2 WILDTYPE INVERTED TERMINAL REPEATS (WTITRS)(WHO)
ZAFTUCLENEGENUM PIRUPARVOVECUM(INN)