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Lentiviral Gene Therapy Approach for Haemoglobinopathies

  • Novel beta and gamma globin gene addition vectors have been generated and evaluated by exvivo erythropoiesis models and mouse models for its therapeutical application in sickle cell disease and beta-thalassaemia
  • In another strategy, an efficient lentiviral vector for the erythroid cell specific expression of BCL11A shRNA has been generated. This vector has been evaluated by the ex-vivo erythropoiesis of transduced CD34+ cells and by transplantation of these cells in NSG and NBSGW mice for testing the potential of the vector to increase foetal haemoglobin (HbF).

Post Author: cscr-nahd