Сесикленеген косапарвовек

Sesiclenegene cosaparvovec

МНН

Rec. INN (наименование, зарегистрированное ВОЗ)

CAS

2379806-98-9

Химическое название

A non-replicating self-complementary adenoassociated virus (scAAV) vector encoding human ceroid-lipofuscinosis neuronal protein 6.
A recombinant non-replicating self-complementary adeno-associated virus serotype 9 (scAAV9) vector, encoding human ceroid-lipofuscinosis neuronal protein 6 (CLN6), under the control of a hybrid CMV enhancer/chicken-beta-actin promoter and a bovine growth hormone (bGH) polyA sequence, with an SV40 intron 5' to the transgene, and flanked by AAV2 inverted terminal repeats (ITRs). The vector genome is a head-to-head, self-complementary dimer, with the vector genome cassette located 5′ of the mutated internal inverted terminal repeat (Δ-ITR) in a reverse complementary orientation and 3′ of the Δ-ITR in a forward orientation.

Иностранные названия

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