autologous CD34+ cells transduced with FACC retroviral vector

This ex vivo gene therapy is designed for the treatment of Fanconi anemia complementation group C (FA-C), a rare genetic disorder characterized by bone marrow failure and DNA repair defects. The treatment involves the isolation of autologous CD34+ hematopoietic stem and progenitor cells from the patient, which are then transduced ex vivo with a retroviral vector (typically the MFG or LXSN backbone) carrying a functional human FANCC cDNA. Once reinfused into the patient, these modified cells are intended to engraft in the bone marrow and produce progeny with a restored DNA repair mechanism. The FANCC protein is a critical component of the Fanconi anemia (FA) core complex, which regulates the monoubiquitination of FANCD2 and FANCI, a key step in the repair of DNA interstrand crosslinks. By restoring this pathway, the therapy aims to provide a survival advantage to hematopoietic cells and mitigate the progression of aplastic anemia.

Other names

FANCC gene therapyautologous CD34+ cells transduced with FANCCretrovirus-mediated FACC gene transfer