Gene therapy of inherited bleeding disorders Haemophilia A – Lentiviral vector based FVII gene addition in phase 1 clinical trial Haemophilia B – Adeno associated vector based FIX gene addition Gene therapy of inherited haemoglobin disorders – Beta thalassemia major and sickle cell disease Beta and gamma gene addition through lentiviral vectors Modulation of BCL11A gene through lentiviral vector based delivery of shRNA Modulation of BCL11A/other targets for HbF elevation using gene editing technologies – CRISPR-Cas9, base and prime editing Application of gene editing technologies for correction of specific mutations Gene therapy for HIV infection through gene edited haematopoietic stem cells
