Ровоктокоген дурпарвовек

Rec. INN (наименование, зарегистрированное ВОЗ)

a recombinant intronless non-replicating adeno-associated virus 2 vector (AAV2) with a bio-engineered capsid derived by DNA shuffling of 10 AAV capsid sequences (AAV1, 2, 3B, 4, 5, 6 8, 9, avian and bovine AAV) followed by in vivo selection in Fah−/−/Rag2−/−/Il2rg−/− (FRG) mice partially repopulated with human hepatocytes. The vector has AAV2 genomic inverted terminal repeats (ITR) and encodes a human B-domain-deleted blood coagulation factor VIII (hF8, factor VIII, FVIII) under the control of a modified transthyretin (TTRm) promoter.

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