Ванглусаген энсипарвовек
Vanglusagene ensiparvovecМНН
Rec. INN (наименование, зарегистрированное ВОЗ)
Химическое название
A recombinant liver-directed adeno-associated virus vector expressing human acid alpha-glucosidase (GAA).
A recombinant liver-directed adeno-associated virus serotype rh74 (AAVrh74) vector that contains a bioengineered capsid derived from AAVrh74 by four amino-acid substitutions and a codon-optimized transgene expression cassette to drive hepatic synthesis and secretion of the lysosomal enzyme human acid alpha-glucosidase (GAA, α-1,4- glucosidase, acid maltase) via an apolipoprotein E hepatic control region 1 (ApoE HCR1) enhancer/ human alpha 1-antitrypsin (hAAT) promoter, a modified human beta-globin intron 2 (HBB2) and a bovine growth hormone (bGH) polyA signal sequence, flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs).
A recombinant liver-directed adeno-associated virus serotype rh74 (AAVrh74) vector that contains a bioengineered capsid derived from AAVrh74 by four amino-acid substitutions and a codon-optimized transgene expression cassette to drive hepatic synthesis and secretion of the lysosomal enzyme human acid alpha-glucosidase (GAA, α-1,4- glucosidase, acid maltase) via an apolipoprotein E hepatic control region 1 (ApoE HCR1) enhancer/ human alpha 1-antitrypsin (hAAT) promoter, a modified human beta-globin intron 2 (HBB2) and a bovine growth hormone (bGH) polyA signal sequence, flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs).
Структура
Иностранные названия
- Vanglusagenum ensiparvovecum (латинское)
- Vanglusagene ensiparvovec (английское)
- Vanglusagen ensiparvovec (немецкое)
- Vanglusagène ensiparvovec (французское)
- Vanglusagén ensiparvovec (испанское)
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