EMERGING PERSPECTIVES ON IN VIVO GENE THERAPY

Michela Milani, project leader SR-TIGET

In vivo gene therapy is rapidly emerging as a potential transformative approach for the treatment of genetic disorders, offering the potential for long-lasting therapeutic benefits through the delivery of engineered viral-derived vectors, used as carriers for the corrective genetic material, directly into the bloodstream to reach the patient’s cells in vivo. This seminar will provide an overview of the current landscape of in vivo gene therapy, highlighting key advances, clinical milestones, and the technological platforms driving progress in the field. Dr. Milani will also present her recent research on the use of lentiviral vectors for in vivo gene delivery, discussing their unique advantages, challenges, and therapeutic potential in metabolic and hematological diseases.