MFGS-gp91phox

MFGS-gp91phox is a first-generation gammaretroviral gene therapy vector designed for the treatment of X-linked chronic granulomatous disease (X-CGD). The vector is based on the MFG backbone (a Moloney murine leukemia virus-derived vector) and carries the cDNA for the human *CYBB* gene, which encodes the gp91phox protein, the heavy chain subunit of the phagocyte NADPH oxidase system. In clinical application, autologous CD34+ hematopoietic stem cells (HSCs) are harvested from the patient and transduced ex vivo with the MFGS-gp91phox vector using either amphotropic or RD114 envelopes. After the patient receives nonmyeloablative conditioning (typically busulfan), the corrected HSCs are infused back into the patient. These cells engraft and differentiate into neutrophils that express functional NADPH oxidase, restoring the oxidative burst necessary to fight bacterial and fungal infections. While early trials demonstrated clinical benefits in resolving refractory infections, long-term efficacy was often limited by low marking or silencing, leading to the development of second-generation lentiviral vectors.

Other names

amphotropic MFGS-gp91phoxRD114-pseudotyped MFGS-gp91phoxRD-114-pseudotyped MFGS-gp91phoxRD 114-pseudotyped MFGS-gp91phoxMFG-gp91phoxMFG-gp-91phoxMFG-gp 91phox