G1XCG

G1XCG is an investigational ex vivo gene therapy developed by researchers at Johann Wolfgang Goethe-University for the treatment of X-linked chronic granulomatous disease (X-CGD). X-CGD is a primary immunodeficiency caused by mutations in the *CYBB* gene, which encodes the gp91phox subunit of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase complex. The absence of functional gp91phox prevents phagocytes from generating the reactive oxygen species necessary for killing bacteria and fungi. G1XCG consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been genetically modified ex vivo using a self-inactivating (SIN) gammaretroviral vector. This vector delivers a functional copy of the human *CYBB* cDNA, driven by a myeloid-specific promoter. The goal is to restore NADPH oxidase activity in the patient's neutrophils and monocytes following re-infusion of the corrected cells, thereby providing long-term protection against life-threatening infections.

Other names

autologous CD34+ cells transduced with G1XCG vectorSIN-gammaretroviral vector-transduced CD34+ cells