GALV MFGS-gc transduced autologous CD34+ cells

GALV MFGS-gc transduced autologous CD34+ cells is an investigative ex vivo gene therapy developed for the treatment of X-linked severe combined immunodeficiency (X-SCID), also known as SCID-X1. The therapy involves harvesting a patient's own CD34+ hematopoietic stem cells and transducing them with an MFGS retroviral vector pseudotyped with the Gibbon Ape Leukemia Virus (GALV) envelope protein. The vector carries a functional copy of the human interleukin-2 receptor subunit gamma (IL2RG) gene, which encodes the common gamma chain (γc) protein. Following transduction, the modified cells are re-infused into the patient. These cells are intended to engraft in the bone marrow and differentiate into functional T cells, Natural Killer (NK) cells, and B cells that express the γc protein, thereby restoring critical cytokine signaling pathways and immune function. While clinically effective in early trials, this specific retroviral approach was associated with insertional mutagenesis, leading to the development of more advanced lentiviral and self-inactivating vector designs.

Other names

Autologous CD34+ cells transduced with MFGS-gamma c retroviral vectorIL2RG retroviral gene therapyIL-2RG retroviral gene therapyIL 2RG retroviral gene therapyGALV-pseudotyped retroviral vector-mediated gene transfer of IL2RG