Gene transfer into stem cell

Gene transfer into stem cell


Head of Unit

Giuliana Ferrari


Unit major interests are in the field of hematopoietic stem cell biology and gene transfer to treat genetic diseases. Gene therapy is a therapeutic technique by which a functioning gene is inserted into the somatic cells of a patient to correct a genetic error or to provide a new function to the cell. Since 1989 the unit pioneered the use of viral vector-mediated gene transfer for gene therapy of human genetic diseases.

Research activity

The group developed lentiviral vectors for the transcriptionally regulated expression of human beta-globin to correct hemoglobinopathies and they are leading a clinical trial of gene therapy for beta-thalassemia. Molecular studies will provide insight into human erythropoiesis and hematopoiesis.

Other projects include the study of vector-genome interaction, hematopoietic stem cell biology, hematopoietic stem cell-niche interactions and molecular control of erythropoiesis.