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PIVODISGENE PARVEC

UNII:NVQ25YNH8P
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:PIVODISGENE PARVEC
GSRS Full Record:

  • 2648631-52-9
  • PIVODISGENE PARVEC [USAN]
  • Pivodisgene Parvec
  • RGX-202 (AAV8.Spc5-12.Microdystrophin)
  • Recombinant replication-defective adeno-associated virus 8 vector RGX-202 synthetic promoter Spc5-12 plus human codon-optimized microdystrophin variant ABD1-H1-R1-R2-R3-H3-R24-H4-CR-CT194 plus synthetic polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks-specifying
  • pivodisgene parvec [INN]
  • recombinant, non-replicating adeno-associated virus serotype 8 (rAAV8) vector, encoding a codon-optimised miniaturised version of human dystrophin (microdystrophin variant ABD1-H1-R1-R2-R3-H3-R24-H4-CR-CT194), under control of a synthetic muscle-specific promoter (SPc5-12) and a small synthetic polyadenylation signal; flanked by AAV2 inverted terminal repeats. The expressed microdystrophin variant contains the actin-binding domain 1 (ABD1), four rod domains (R1-3, R24), three hinge domains (H1, H3, H4) and a cysteine rich (CR) domain; it also features the proximal portion of the C-terminal domain, amino acids 3361-3554 (CT194)



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