San Raffaele Telethon Institute for Gene Therapy

Gene transfer into stem cell

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Head of Unit

Giuliana Ferrari

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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.