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CIRACIGENE GOLPARVOVEC

UNII:DM5GA58Q2Z
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:CIRACIGENE GOLPARVOVEC
GSRS Full Record:
DM5GA58Q2Z
i

  • 2847055-64-3
  • A recombinant, self-complementary non-replicating adeno-associated virus vector with Olig001 capsid (rAAV-Olig-001) encoding codon-optimized human aspartoacylase (ASPA, ASP, ACY2)
  • CIRACIGENE GOLPARVOVEC [USAN]
  • Capsomere, recombinant adeno-associated virus clone rAAV-Olig-001 viral capsid protein VP1-VP2-VP3 complex (1:1:10), hexacontamer, complex with DNA (recombinant replication-defective adeno-associated virus vector cytomegalovirus enhancer plus chicken β-actin gene hybrid promoter plus human aspartoacylase codon-optimized gene plus bovine somatotropin gene polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks) (1:1)
  • Ciracigene golparvovec
  • MYR-101
  • ciracigene golparvovec [INN]
  • recombinant, self-complementary non-replicating adenoassociated virus vector with Olig-001* capsid (rAAV-Olig-001) encoding codon-optimized human aspartoacylase (ASPA, ASP, ACY2) under control of a cytomegalovirus (CMV) enhancer/chicken β-actin (CBh) hybrid promoter and terminated by a bovine growth hormone polyadenylation signal, flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs). The vector genome is a head-to-head, selfcomplementary dimer, with the vector genome cassette located 5' of the mutated internal inverted terminal repeat (Δ-ITR) in a reverse complementary orientation and 3' of the Δ-ITR in a forward orientation (*The Olig-001 capsid is a chimeric mixture of AAV1, 2, 6, 8 and 9 that facilitates entry into oligodendrocytes)




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