Атидарсаген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

Autologous CD34+ hematopoietic stem and progenitor cells transduced ex vivo with a replication-incompetent lentiviral vector encoding human arylsulfatase A (ARSA). Autologous CD34+ hematopoietic stem and progenitor cells (HSPC), obtained from bone marrow (BM) or mobilised peripheral blood (mPB), transduced ex vivo with a replication-incompetent pseudotyped HIV-1 based lentiviral vector encoding the human arylsulfatase A (ARSA) transgene under the control of a human phosphoglycerate kinase (PGK) 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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