Мозафанкоген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

autologous CD34+ cells obtained from mobilised peripheral blood, transduced with a self-inactivating lentiviral vector expressing human Fanconi anemia complementation group A (FANCA) 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 ψ packaging signal, a Rev response element (RRE), a central polypurine tract (cPPT) sequence and the Woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The vector is pseudotyped with the envelope glycoprotein (G) of vesicular stomatitis virus (VSV). The CD34+ cell population was enriched using immunomagnetic microbeads, and the cells cultured in bags coated with a fragment of recombinant human fibronectin in the presence of medium containing stem cell factor (SCF), FMS-related tyrosine kinase 3 ligand (Flt-3L), thrombopoietin (TPO), interleukin 3 (IL-3)) and the TNF-α inhibitor etanercept. Myeloid differentiated cells generated from the transduced CD34+ cells are able to form colonies in the presence of mitomycin (mitomycin-C, MMC).

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