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Review
Gene therapy in India: A focus
SARVANI CHODISETTY and EVERETTE JACOB REMINGTON NELSON*
Gene Therapy Laboratory, School of Biosciences and Technology, VIT University, Vellore 632 014, India
*Corresponding author (Email, [email protected])
Gene therapy refers to the treatment of genetic diseases using normal copies of the defective genes. It has the potential to cure any genetic disease with long-lasting therapeutic benefits. It remained an enigma for a long period of time, which was followed by a series of setbacks in the late 1990s. Gene therapy has re-emerged as a therapeutic option with reports of success from recent clinical studies. The United States and Europe has been pioneers in this field for over two decades. Recently, reports of gene therapy have started coming in from Asian countries like China, Japan and Korea. This review focuses on the current status of gene therapy in India.
[Chodisetty S and Nelson EJR 2014 Gene therapy in India: A focus. J. Biosci. 39 537541] DOI http://dx.doi.org/10.1007/s12038-014-9431-2
Web End =10.1007/s12038-014-9431-2
Gene therapy remained an enigma for a long time, until early reports of gene therapy started emerging in the 1990s. Gene therapy had some major initial setbacks starting with the death of Jesse Gelsinger, an 18-year-old boy who was suffering from ornithine transcarbamylase deficiency. He was reported to have died from multiple organ failure that was caused by a severe immune reaction in response to the use of first-generation adenoviral vectors (Raper et al. 2003). This was followed by the death of two patients who were treated for X-linked severe combined immunodeficiency (X-SCID) using retroviral vectors. They died of leukaemia which had developed as a result of an exponential T cell proliferation from a single clone (Hacein-Bey-Abina et al. 2003). These two failures led to an immediate halt to all gene therapy clinical trials worldwide, especially the ones involving viral vectors. It was not until the last decade that gene therapy for monogenic disorders like severe combined immunodeficiency (SCID), Parkinsons disease, Lebers congenital amaurosis, etc., made its way through various stages of clinical trials. Since then, there has been a steady increase in the number of gene therapy clinical trials around the world.
Several gene therapy clinical studies carried out in different parts of the world after 2008 have clearly demonstrated...