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LIBENUCGENE PARVEC

UNII:EMR895S25C
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:LIBENUCGENE PARVEC
GSRS Full Record:

  • 3044760-95-1
  • AAVrh79.TBG.M2PCSK9.WPRE.bGH
  • DNA (recombinant replication-deficient adeno-associated virus serotype rh79 vector ECUR-506A human α1-microglobulin-bikunin precursor enhancer plus human thyroxine binding globulin promoter plus simian virus 40 nuclear localization signal peptide fusion protein with synthetic meganuclease M2PCSK9 codon-optimized transgene plus woodchuck hepatitis virus post-transcriptional regulatory element WPRE plus bovine growth hormone polyadenylation signal plus inverted terminal repeat flanks)
  • ECUR-506A
  • LIBENUCGENE PARVEC [USAN]
  • Libenucgene Parvec
  • Recombinant non-replicating adeno-associated virus serotype rh79 (rAAVrh79) vector encoding a fusion protein comprising a simian virus 40 (SV40) nuclear localization signal (NLS) joined to an engineered meganuclease (M2PCSK9) targeting a specific 22-base pair sequence in exon 7 of the human proprotein convertase subtilisin/kexin type 9 (PCSK9) gene. Expression of the transgene is under the control of a human thyroxine binding globulin (TBG) promoter and two copies of an alpha 1-microglobulin/bikunin enhancer element, a Woodchuck hepatitis virus post-transcriptional regulatory element (WPRE) and a bovine growth hormone polyadenylation signal. The expression cassette is flanked by AAV2 inverted terminal repeat
  • libenucgene parvec [INN]



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