Арвенакоген санпарвовек

Prop. INN (наименование, предложенное ВОЗ)

2891724-48-2

recombinant non-replicating adeno-associated virus serotype 5 (AAV5) vector encoding a codon-optimized gain of function variant (Padua variant, R338L) of human coagulation factor IX (FIX), under the control of a murine liver-specific transthyretin (TTR) promoter and human growth hormone polyadenylation signal, flanked by AAV2 inverted terminal repeats. A human beta-globin intron has been inserted between the promoter and the FIX coding region. The virus vector also contains mutations in the viral AAV5 capsid protein VP1 (S2A and T711S)

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