Gene therapy advance

New Indian technology licensed to US company

By T V Jayan
Last Updated: Saturday 04 July 2015

researchers at the University of Delhi have given a us pharmaceutical company the licence to use a novel technique developed by them that could help commercialise gene therapy. The gene delivery technology developed by A N Maitra and his associates at the university's Department of Chemistry uses calcium phosphate nanoparticles to deliver specific engineered genes to infected cells. This can help in the treatment of many diseases like cancer, cardiovascular diseases and Parkisnson's disease. In the field of pharmaceuticals sciences, it is arguably the first ever technology developed at an Indian university to be licensed to a foreign firm.

The basic concept of gene therapy is to introduce a gene whose product has the ability to cure or slow down the progression of disease into cells. Typically, scientists use harmless viruses for shuttling such genes to cells. "Though such tamed viruses are efficient in delivering the therapeutic genes to the cells, these methods are not fully acceptable due to dangers involved in introducing viruses into the cells, however harmless they are to be," says Maitra. A number of non-viral carriers have also been attempted, but most have failed. The speciality of this new technique is that it is a non-viral technique that seems to work.

The National Research Development Corporation, the state arm to commercialise technologies developed through public-funded research projects, licensed the new technology to the us pharmaceutical firm American BioSciences for an aggregate payment of us $345,000 in November 2004. The company will also pay a royalty of four per cent, to be shared between the government and the university, once they commercialise the technology. The Rs 67 lakh research project was funded by the Union government's Department of Science and Technology.

The low efficiency in non-viral vectors stems from their inability to cross various layers inside a cell and reach its nucleus. But when a therapeutic gene is delivered, encapsulated in calcium phosphate nanoparticles, it manages to reach the nucleus. This is because the normal human body temperature of 37 c is also the temperature at which calcium phosphate dissolves in the acids contained in the endosome (the intracellular compartment isolated from the rest of the cell). The endosome expands as a result and bursts, helping the gene to bypass the lysomes, a set of minute bodies whose enzymes are involved in localised digestion. The therapeutic dna crosses the cytosol, the final barrier before the nucleus, and enters the nucleus with the help of divalent metal ions in calcium phosphate.

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