lentiviral vector transduced CD34+ cells

Lentiviral vector transduced CD34+ cells are autologous or allogeneic human hematopoietic stem and progenitor cells (HSPCs) that have been genetically modified ex vivo using a lentiviral vector to introduce a therapeutic gene. The process involves isolating CD34+ HSPCs from bone marrow or mobilized peripheral blood, transducing them with a clinical-grade lentiviral vector encoding the desired gene (such as for correction of monogenic disorders), and then reinfusing the modified cells into the patient. These engineered HSPCs can engraft in the bone marrow and give rise to multiple blood lineages expressing the therapeutic protein. This approach is being developed for diseases such as Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, sickle cell anemia, thalassemia, and other inherited immunohematological disorders[2][6][1]. The mechanism of action is based on stable integration of the corrective gene into long-term repopulating HSCs via lentiviral vectors—enabling durable expression in differentiated progeny[1][2].

Other names

lentiviral vector-transduced CD34-positive cellsLV-transduced CD34+ cellsgene-modified CD34+ hematopoietic stem/progenitor cells