Дальнакоген понпарвовек

Prop. INN (наименование, предложенное ВОЗ)

2770683-67-3

recombinant, self-complementary, non-replicating adeno- associated virus, synthetic liver-tropic serotype 843 (rAAV843) vector, encoding a codon-optimised (CpG reduced) gain of function variant (Padua variant; R338L) of human coagulation factor IX (FIX), under control of a synthetic liver-specific Lxp2.1 promoter and a synthetic polyadenylation (polyA) sequence derived from the rabbit beta-globin gene polyA signal. The expression cassette also contains a human DNA-dependent RNA polymerase II gene intron, a Kozak sequence, a human immunoglobulin heavy chain variable region gene intron inserted into the middle of the FIX coding region, and an enhancer derived from the promoter region of human alpha-1-antitrypsin. The transgene is flanked by adeno- associated virus 2 (AAV2) inverted terminal repeats (ITRs)

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