autologous CD34+ cells transduced with G2SCID vector

Autologous CD34+ cells transduced with the G2SCID vector is an ex vivo gene therapy designed to treat X-linked severe combined immunodeficiency (SCID-X1), a life-threatening primary immunodeficiency. The therapy involves harvesting hematopoietic stem and progenitor cells (CD34+ cells) from the patient and modifying them using a self-inactivating (SIN) lentiviral vector (rHIV_IL2RGcoG2SCID). This vector delivers a codon-optimized functional copy of the *IL2RG* gene, which encodes the common cytokine receptor gamma chain (γc). The γc subunit is essential for the formation of cytokine receptors (IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21) that drive the development and maturation of T-lymphocytes, B-lymphocytes, and natural killer (NK) cells. Following low-dose busulfan conditioning to facilitate bone marrow engraftment, the genetically corrected cells are infused back into the patient. These cells kemudian proliferate and differentiate into functional immune cells, potentially providing a durable, curative restoration of immunity. This therapy is primarily targeted at newly diagnosed infants or patients who have failed prior hematopoietic stem cell transplantation and lack a matched sibling donor.

Other names

G2SCID lentiviral vector transduced CD34+ cellsrHIV_IL2RGcoG2SCID-transduced CD34+ cellsautologous CD34+ cell transduced with G2SCID vector