MFGS-p47phox

MFGS-p47phox is an ex vivo gammaretroviral gene therapy developed for the treatment of the p47phox-deficient form of Chronic Granulomatous Disease (CGD). The therapy involves the transduction of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) with a Moloney murine leukemia virus-based gammaretroviral vector (MFGS) encoding the human NCF1 (p47phox) gene. Once the genetically modified cells are re-infused into the patient, they engraft and differentiate into myeloid cells, such as neutrophils and macrophages, which express the functional p47phox protein. This protein is an essential cytosolic subunit of the NADPH oxidase complex; its restoration allows the cells to generate the reactive oxygen species (the "oxidative burst") required for microbial killing. While early clinical trials conducted by the NIH demonstrated proof-of-concept for biochemical correction, the program faced challenges including low levels of long-term gene expression and the inherent risks of insertional mutagenesis associated with first-generation gammaretroviral vectors. Consequently, this specific construct has largely been superseded by self-inactivating lentiviral and gene-editing platforms in contemporary clinical research.

Other names

MFG-S-p47phoxMFG-S-p-47phoxMFG-S-p 47phoxGammaretroviral-p47phoxGammaretroviral-p-47phoxGammaretroviral-p 47phoxMFGS-NCF1 gene therapyMFGS-NCF-1 gene therapyMFGS-NCF 1 gene therapyMFGS-p47-phoxMFGS-p-47-phoxMFGS-p 47-phox