Воксералгаген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

Autologous CD34+ hematopoietic stem cells (HSCs) transduced ex vivo with a self-inactivating lentiviral vector encoding human alpha-galactosidase A (GLA). Autologous CD34+ hematopoietic stem cells (HSCs) obtained by apheresis, transduced with a nonreplicating, self-inactivating lentiviral vector encoding codon-optimized human alpha-galactosidase A (GLA) under the control of the human elongation factor 1 alpha short (EFS-1 alpha) promoter and a modified woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The vector genome also contains a packaging signal, a partial gag sequence, a Rev response element (RRE), a central polypurine tract (cPPT) and a Kozak sequence.

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