Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells

Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells are autologous or allogeneic human hematopoietic stem cells (CD34+) that have been genetically modified ex vivo to express a functional subunit of the phagocyte NADPH oxidase complex, most commonly the gp91phox protein encoded by the CYBB gene. This therapy is designed for patients with chronic granulomatous disease (CGD), particularly X-linked CGD, which results from mutations in genes encoding components of the NADPH oxidase system and leads to defective microbial killing by phagocytes. The modification is typically achieved using a lentiviral or retroviral vector carrying a codon-optimized cDNA for gp91phox, enabling corrected myeloid progeny to restore superoxide production and antimicrobial function. The product is reinfused into patients following conditioning chemotherapy[1][2][4].

Other names

gp91phox-transduced CD34+ hematopoietic stem cellsCYBB gene-modified CD34+ HSCsG1XCGD Modified Autologous BM CD34 cellsG-1XCGD Modified Autologous BM CD34 cellsG 1XCGD Modified Autologous BM CD34 cells