autologous CD34+ cells transduced with SINfes.gp91s gammaretroviral vector

This is an investigational ex vivo gene therapy consisting of autologous hematopoietic stem and progenitor cells (CD34+ cells) that have been genetically modified using a self-inactivating (SIN) gammaretroviral vector. The vector, designated SINfes.gp91s, carries a functional copy of the human CYBB gene (encoding the gp91phox protein) under the transcriptional control of a myelospecific promoter (human c-fes). The therapy is designed to treat X-linked Chronic Granulomatous Disease (X-CGD) by restoring the expression of the Cytochrome b-245 heavy chain, a critical component of the NADPH oxidase complex. By reintroducing these modified cells into the patient via intravenous infusion, the therapy aims to produce functional neutrophils capable of generating reactive oxygen species, thereby restoring immune defense against bacterial and fungal infections. Although this SIN-gammaretroviral approach was developed to improve safety over earlier gammaretroviral designs, clinical development was largely superseded by lentiviral vector technologies.

Other names

SIN-gammaretroviral gene therapy for X-CGDSelf-inactivating gammaretroviral vector-transduced CD34+ cells