Ниматпаген парипарвовек

Rec. INN (наименование, зарегистрированное ВОЗ)

2773545-20-1

recombinant, non-replicating adeno-associated virus serotype 3B (rAAV3B) vector, encoding a shortened version of the human copper-transporting ATPase 2 (ATP7B) (also known as ATPase copper-transporting beta, Wilson disease-associated protein) which is devoid of the first four of the six metal binding sites (MBS) (mini-ATP7B), under control of a liver specific human alpha-1 antitrypsin (AAT) promoter and a synthetic polyadenylation sequence derived from the polyadenylation signal of the rabbit ß-globin gene, and flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs)

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