Зафтукленеген пирупарвовек

Rec. INN (наименование, зарегистрированное ВОЗ)

2567881-78-9

recombinant, self-complementary (dimeric), non- replicating adeno-associated virus serotype 9 (scAAV9) vector, encoding codon-optimised human palmitoyl-protein thioesterase 1 (PPT1, CLN1), under the control of a synthetic promoter consisting of a cytomegalovirus (CMV) enhancer/chicken β-actin (CBA) promoter and a hybrid intron derived from chicken β-actin and minute virus of mice (MVM) introns, followed by a Kozak sequence, and terminated by a bovine growth hormone (bGH) polyadenylation (polyA) signal; the vector genome contains a mutated adeno-associated virus serotype 2 (AAV2) inverted terminal repeat (mITR) and is flanked by AAV2 wild- type inverted terminal repeats (wtITRs).

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