Лонимекген ренпарвовек

Prop. INN (наименование, предложенное ВОЗ)

2856375-48-7

recombinant, self-complementary (dimeric), non- replicating adeno-associated virus serotype 9 (scAAV9) vector encoding a miniaturised form of human methyl-CpG binding protein 2 (MECP2) containing the methyl-CpG-binding domain (MBD) and the NCoR/SMRT (silencing mediator of retinoic acid and thyroid hormone receptor) interaction domain (NID), under the control of a fragment of the mouse MECP2 promoter. The 3' untranslated region is designed to decrease protein expression through RNA interference by containing a microRNA (miR) Responsive Auto-Regulatory Element (miRARE) inserted into the replication dependent histone 1 (RDH1) polyadenylation region. The construct is flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs)

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