Азамидуген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

autologous CD34+ cells obtained from mobilised peripheral blood, transduced with a self-inactivating lentiviral vector expressing human alpha-L-iduronidase (IDUA) under the control of a human phosphoglycerate kinase (PGK) promoter; the construct is flanked by 5' and 3' long terminal repeats (LTRs) and also contains a HIV-1 ψ packaging signal, a Rev response element (RRE), a central polypurine tract (cPPT) sequence and the Woodchuck hepatitis virus post-transcriptional regulatory element (WPRE). The vector is pseudotyped with the envelope glycoprotein (G) of the vesicular stomatitis virus (VSV). The CD34+ cell population was enriched using immunomagnetic microbeads, and the cells cultured in the presence of media containing FMSrelated tyrosine kinase 3 ligand (Flt-3L), interleukin 3 (IL-3), thrombopoietin (TPO) and stem cell factor (SCF). The final substance contains ≥70% CD34+ cells.

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