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PASEMNOGENE GLEPARVOVEC

UNII:2Q4YZ4G35L
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:PASEMNOGENE GLEPARVOVEC
GSRS Full Record:

  • 2913126-89-1
  • Capsomere, recombinant adeno-associated virus 9 viral capsid protein VP1 [422-tryptophan,446-phenylalanine] complex with viral capsid protein VP2 [285-tryptophan,309-phenylalanine] complex with viral capsid protein VP3 [220-tryptophan,244-phenylalanine] decamer, hexacontamer, complex with DNA (recombinant adeno-associated virus vector pAAV-SMN1-GeneBeam v2020-03-11 cytomegalovirus enhancer region plus cytomegalovirus promoter region plus human β-globin gene intron fragment plus human survival of motor neuron protein SMN1 codon-optimized gene plus human somatotropin gene polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks) (1:1)
  • Pasemnogene Gleparvovec
  • pasemnogene gleparvovec [INN]
  • recombinant non-replicating adeno-associated virus serotype 9 (AAV9) vector, encoding a codon-optimised human survival of motor neuron 1 (SMN1) gene, under control of the cytomegalovirus (CMV) enhancer/promoter and terminated by a human growth hormone polyadenylation signal, flanked by AAV2 inverted terminal repeats. A human beta-globin intron has been inserted between the promoter and the SMN1 coding region. The virus vector also contains mutations in the viral capsid protein VP1 (F422W and Y446F)



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