Our gene therapy research program involves two major areas at present – The first is directed towards a clinical trial for AAV vector-based gene therapy for haemophilia B in collaboration with Emory University, Atlanta, USA and the Powell Gene Therapy Centre as well as scientists at the University of Florida, Gainesville, USA. Given the success of AAV-based gene therapy reported in the last 4 years, the plan here is to apply a similar yet innovative approach to initiate a clinical trial in India with a novel AAV.
The second part of the gene therapy program involves pre-clinical models for lentiviral vector-based gene therapy through hematopoietic stem cell for the major hemoglobin disorders. This is in collaboration with the Emory University, USA. Lentiviral vectors carrying the beta globin gene are tested in human ex-vivo erythropoietic systems. There has been much progress in this area of work also in the last 1-2 years. Work towards using genome editing technologies towards therapeutic gene corrections for haemoglobin disorders is also being pursued. Other non-vector mediated gene transfer technologies are also being explored. Further details may be accessed at www.cscr.in