Future needs of gene therapy in India

Besides genetic diseases, gene therapy has tremendous scope for treatment of several disorders such as cancer, cardiovascular and neurological disorders. To initiate gene therapy the functional studies should be focussed on (i) design of newer vectors for gene delivery, newer approaches to systemic delivery, (ii) targeting to specific tissues and cells, (iii) stability and duration of expression of gene introduced, (iv) the statutes of the introduced genes in vivo, (v) design of appropriate animal models, (vi) assessment of risk-benefit states, and (vii) an understanding of molecular basis of cellular humoral immune response in case of DNA vaccines (Rangarajan and Padmanaban, 1996).

The need of hours is to start more studies in different systems on various aspects realizing the significance of approaches of gene therapy. Department of Biotechnology, Govt of India has constituted a group of experts to activate this area of research. At present there are only 3-4 laboratories in this country that have started work on this area. At South Campus of Delhi University, in 1994 Bagai and Sarkar have demonstrated the delivery of macromolecules such as toxins, drugs and nucleic acids into viable cultured cells through the use of reconstituted Sendai virus envelop (RSVE). This approach needs to be extrapolated under in vivo conditions. At the Cancer Research Institute (Mumbai) treatment of oral cancer through gene targeting to oral mucosal cells has been demonstrated. In 1995, at the Indian Institute of Science (Bangalore), Prabhu and co-workers have demonstrated the reporter mediated targeting and expression of growth hormone gene in rat liver by using a regulatable promoter (cytochrome P-450 promoter induced by phenobarbitone). In 1996, Rangarajan and co-workers have demonstrated a newer approach to delivering DNA to hepatocytes by perfusion of liver with DNA-lipofectin complex.