Цирациген голпарвовек

Prop. INN (наименование, предложенное ВОЗ)

2847055-64-3

recombinant, self-complementary non-replicating adeno- associated virus vector with Olig-001* capsid (rAAV-Olig-001) encoding codon-optimized human aspartoacylase (ASPA, ASP, ACY2) under control of a cytomegalovirus (CMV) enhancer/chicken β-actin (CBh) hybrid promoter and terminated by a bovine growth hormone polyadenylation signal, flanked by adeno-associated virus 2 (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.
*The Olig-001 capsid is a chimeric mixture of AAV1, 2, 6, 8 and 9 that facilitates entry into oligodendrocytes.

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