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LUNSOTOGENE PARVEC

UNII:P6ZNU67HPU
Formula:
Preferred Substance Name:LUNSOTOGENE PARVEC
GSRS Full Record:
P6ZNU67HPU
i

  • 2907748-12-1
  • CAPSOMERE, RECOMBINANT ADENO-ASSOCIATED VIRUS 1 VIRAL CAPSID PROTEIN VP1-VP2-VP3 COMPLEX (1:1:10), HEXACONTAMER, COMPLEX WITH DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR DB-OTO-5 SYNTHETIC GENE MYO15 PROMOTER REGION PLUS HUMAN OTOFERLIN ISOFORM 5 GENE 5'-FRAGMENT PLUS SYNTHETIC SPLICE DONOR SITE PLUS RECOMBINANT ALKALINE PHOSPHATASE GENE FRAGMENT PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS) (1:1), MIXT. WITH CAPSOMERE RECOMBINANT ADENO-ASSOCIATED VIRUS 1 VIRAL CAPSID PROTEIN VP1-VP2-VP3 COMPLEX (1:1:10) HEXACONTAMER COMPLEX WITH DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR DB-OTO-3 RECOMBINANT ALKALINE PHOSPHATASE GENE FRAGMENT PLUS SPLICE ACCEPTOR REGION PLUS HUMAN OTOFERLIN ISOFORM 5 GENE 3'-FRAGMENT PLUS BOVINE SOMATOTROPIN GENE POLYADENYLATION SIGNAL PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS) (1:1)
  • LUNSOTOGENE PARVEC [INN]
  • TWO RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 1 (RAAV1) VECTORS CONTAINING THE 5' AND 3' PORTIONS, RESPECTIVELY, OF THE WILD-TYPE HUMAN OTOFERLIN (OTOF) ISOFORM 5 GENE. THE 5' VECTOR CONTAINS EXONS 1-20 OF THE OTOF GENE UNDER EXPRESSION CONTROL OF THE HAIR CELL-SPECIFIC MYOSIN 15 (MYO15) PROMOTER, FOLLOWED BY A SPLICE DONOR AND ALKALINE PHOSPHATASE HOMOLOGY REGION 3' TO THE TRANSGENE. THE 3' VECTOR CONTAINS THE ALKALINE PHOSPHATASE HOMOLOGY REGION AND A SPLICE ACCEPTOR, FOLLOWED BY EXONS 21-45, AND 47 OF THE OTOF GENE AND IS TERMINATED BY A BOVINE SOMATOTROPIN GROWTH HORMONE POLYADENYLATION SEQUENCE. BOTH VECTORS ARE FLANKED BY AAV2 INVERTED TERMINAL REPEATS




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