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LUNSOTOGENE PARVEC

UNII:P6ZNU67HPU
Type:INGREDIENT SUBSTANCE
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)
  • DB-OTO
  • LUNSOTOGENE PARVEC [USAN]
  • Lunsotogene Parvec
  • Lunsotogene Parvec-CWHA
  • Otarmeni
  • 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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