SF71-gp91phox transduced CD34+ cells

SF71-gp91phox transduced CD34+ cells is an investigational ex vivo gene therapy developed for the treatment of X-linked Chronic Granulomatous Disease (X-CGD). The therapy utilizes a gammaretroviral vector, specifically SF71 (a derivative of the Spleen Focus Forming Virus/Murine Embryonic Stem Cell Virus hybrid), to deliver a functional copy of the *CYBB* gene into autologous CD34+ hematopoietic stem and progenitor cells. The *CYBB* gene encodes the gp91phox protein, which is the catalytic subunit of the NADPH oxidase complex required for the production of reactive oxygen species in phagocytes. By restoring this enzymatic function, the therapy aims to enable neutrophils to kill bacteria and fungi, thereby preventing the recurrent, life-threatening infections associated with CGD. Clinical trials conducted in the mid-2000s demonstrated high initial rates of biochemical correction; however, the use of this specific gammaretroviral vector with a strong SFFV promoter led to serious adverse events, including insertional mutagenesis and the development of myelodysplastic syndrome or leukemia in several patients, leading to the termination of these early-generation programs.

Other names

SF71-gp91phox-transduced hematopoietic stem cellsSF-71-gp91phox-transduced hematopoietic stem cellsSF 71-gp91phox-transduced hematopoietic stem cellsgammaretroviral gp91phox gene therapySF71gp91phoxSF-71gp91phoxSF 71gp91phox