pCHIM-p47phox

pCHIM-p47phox is a self-inactivating (SIN) lentiviral vector-based gene therapy designed for the treatment of Chronic Granulomatous Disease (CGD) caused by mutations in the *NCF1* gene (p47phox-deficient CGD). The therapeutic construct consists of the human *NCF1* (p47phox) cDNA sequence under the transcriptional control of a myeloid-specific chimeric promoter, pCHIM, which is composed of regulatory elements from the human cathepsin G and c-fes genes. This promoter is designed to restrict the expression of the transgene to the myeloid lineage, specifically neutrophils and monocytes. Developed by researchers at the National Institute of Allergy and Infectious Diseases (NIAID) and University College London (UCL), the therapy involves the ex vivo transduction of autologous CD34+ hematopoietic stem and progenitor cells. Following infusion into the patient, these cells engraft and differentiate into functional myeloid cells that can form a proper NADPH oxidase complex, thereby restoring the antimicrobial respiratory burst and preventing life-threatening bacterial and fungal infections.

Other names

pCHIM-p47pCHIM-p-47pCHIM-p 47pCHIM-p47-PHOXpCHIM-p-47-PHOXpCHIM-p 47-PHOX