FANCC-transduced autologous CD34+ cells

FACC-transduced CD34+ cells are an autologous ex vivo gene therapy designed to treat Fanconi Anemia (FA) patients belonging to complementation group C. The therapy involves harvesting hematopoietic stem and progenitor cells (HSPCs), specifically those expressing the CD34 surface marker, from the patient. These cells are then genetically modified ex vivo using a viral vector (historically retroviral, though lentiviral vectors are more common in modern iterations) to deliver a functional copy of the *FANCC* (Fanconi anemia complementation group C) cDNA. The modified cells are re-infused into the patient with the goal of establishing a population of hematopoietic cells capable of normal DNA repair. The FANCC protein is a vital member of the Fanconi Anemia core complex, which facilitates the repair of DNA interstrand cross-links (ICLs). By restoring *FANCC* expression, the therapy aims to protect the bone marrow from progressive failure and reduce the genomic instability that leads to the severe clinical manifestations of the disease.

Other names

FACC-transduced CD34+ cellsFANCC-transduced CD34+ cellsAutologous CD34+ cells transduced with the FANCC gene