Should we consider gene therapy in severe inherited thrombophilia?: Communication from the ISTH SSC subcommittee on Physiological Anticoagulants and Thrombophilia

The ongoing progress in the use of gene therapy for the treatment of Haemophilia has sparked interest in the world of thrombophilia. We consider which patients with inherited thrombophilias might in particular benefit from such an approach, mindful of the current risk/benefits of current anticoagulation and the need for some very high risk thrombophilias to receive regular plasma products. We review the current approaches to gene therapy. We conclude that with the current limitations of gene therapy, it is not yet time to conduct trials of gene therapy in our inherited thrombophilic patients, but we will continue to monitor the field with great interest.