Нефонадоген вонзупарвовек

Prop. INN (наименование, предложенное ВОЗ)

2779554-99-1

recombinant, non-replicating adeno-associated virus serotype 2 (rAAV2) vector, encoding a mitochondrial targeting sequence from the human heme A farnesyltransferase gene (COX10) fused to codon- optimised human mitochondrial NADH dehydrogenase subunit 1 (MT-ND1, NADH1, ND1; also known as NADH- ubiquinone oxidoreductase chain 1), under control of the cytomegalovirus (CMV) immediate early enhancer/promoter and the simian virus 40 (SV40) polyadenylation signal. The 5' untranslated region (UTR) also contains a Macacine herpesvirus 3 chimeric intron, 3' of the CMV enhancer/promoter and the Kozak sequence, and the 3' untranslated region comprises the COX10 3' UTR. The expression cassette is flanked by AAV2 inverted terminal repeats

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