Этуветидиген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

autologous CD34+ hematopoietic stem and progenitor cells (HSPC), obtained from bone marrow or mobilised peripheral blood. The cells are transduced ex vivo with a self-inactivating (SIN), replication-incompetent human immunodeficiency virus 1 (HIV-1) based lentiviral vector encoding the Wiskott-Aldrich syndrome (WAS) protein under the control of the human WAS gene 1.6 kb promoter. The vector is pseudotyped with the vesicular stomatitis virus (VSV) envelope glycoprotein. The vector genome also contains a mutated Woodchuck hepatitis virus post-transcriptional regulatory element (WPRE), a fragment of the HIV-1 gag, the HIV-1 Rev response element (RRE), the HIV-1 central polypurine tract (cPPT) and a fragment of nef. The CD34+ cell population corresponds to an heterogenous population of stem and progenitor cells, some expressing the CD90 and/or CD133 markers.

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