Gene therapy of haemoglobinopathy: An Italy-USA project.

The article presents the collaborative Italy-U.S.. project study for gene therapy of hematopoietic stem cells (HSCs). The study aims to produce globin lentiviral (LV) vectors with improved titer gene transfer and expression to improve efficiency and safety of the TNS9 lentiviral vector for gene therapy of beta-thalassemia major. It also demonstrates the critical aspect of gene therapy which is the efficiency of the systems of gene transduction.