Лецилимоген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

autologous CD34+ cells obtained by apheresis from mobilised peripheral blood of patients who received a prior hematopoietic stem cell transplant, transduced ex vivo with a self-inactivating lentiviral vector, encoding codon-optimized human interleukin-2 receptor subunit gamma (IL2RG) also known as common gamma chain (γc), driven by a short version of the EF1α promoter.
The vector contains human immunodeficiency virus (HIV) central polypurine tract sequences, the HIV Rev response element (RRE) and is flanked by HIV long terminal repeats. The vector also contains a 400 bp insulator fragment from the chicken β-globin locus inserted into the deleted U3 region of the HIV long terminal repeat followed by a polyadenylation sequence derived from the rabbit β-globin gene. The vector is pseudotyped with the envelope glycoprotein (G) of vesicular stomatitis virus (VSV).
The suspension is enriched for CD34+ cells using magnetic bead separation. The cells are overnight cultured in media containing human serum albumin (1%), human stem cell factor (SCF), human thrombopoietin protein (TPO), and human Flt3 ligand (Flt3L) prior to vector transduction.

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