Тидагиксаген дерксепарвовек

Rec. INN (наименование, зарегистрированное ВОЗ)

2644621-85-0

recombinant, self-complementary (dimeric), non- replicating adeno-associated virus serotype 9 (scAAV9) vector encoding codon-optimised human gigaxonin (giant axonal neuropathy [GAN] gene, Kelch-like protein 16) under the control of the artificial JeT promoter (built from fragments of the viral simian virus 40 [SV40] early promoter and the human beta- actin and ubiquitin C promoters), terminated by a synthetic polyadenylation sequence, and flanked by AAV2 inverted terminal repeats (ITRs).

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