Крозигалкоген омликспарвовек

Rec. INN (наименование, зарегистрированное ВОЗ)

recombinant, non-replicating adeno-associated virus serotype hu68 (AAVhu68) vector encoding codon- optimised human galactosylceramidase (GALC), under control of a cytomegalovirus immediate early enhancer/chicken β-actin promoter (the CB7 promoter) plus a chimeric intron, consisting of a chicken β-actin splice donor and a rabbit β-globin splice acceptor, terminated with a rabbit β-globin polyadenylation signal, flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs).

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