We have shown in the laboratory that transfer of the FA-C gene will improve the growth of early bone marrow progenitor cells. This has prompted research to understand and improve the transfer of genes into human blood-forming cells.
Viruses appear to be the most efficient means of transferring genes into cells.
Retroviral vectors have been extensively studied and most frequently used in clinical gene therapy trials. We have demonstrated that this vector carrying the FA-C gene can also infect early hematopoietic progenitor cells in the laboratory, as well as in mice, and more recently, primates. Work is currently focused on developing strategies to improve gene transfer for clinical trials.