Kanpur, The Indian Institute of Technology (IIT) in Kanpur has licensed a pioneering technology to Reliance Life Sciences Pvt. Ltd. that has the potential to revolutionise the field of gene therapy, especially for many genetic eye diseases. There are many inherited disorders caused by a faulty gene.
“Gene Therapy” is a way to replace the faulty gene with a functional version of the gene to treat such disorders.
This marks the first time that a gene therapy related technology has been developed and transferred from an academic institution to a company in India. The gene therapy technology from IIT Kanpur will be further developed as an Indigenous Product by Reliance Life Sciences, said an official release from IIT-K.
Developed by Prof Jayandharan Giridhara Rao and Shubham Maurya from the Department of Biological Sciences and Bioengineering (BSBE), IIT Kanpur, the patented technology modifies the gene of an organism to treat a hereditary disorder.
In this case, the site refers to a specific location on an Adeno-associated virus (AAV) (viral vector) used for gene therapy. The technology modifies this location to optimize its ability to deliver genes to the affected cells and improve its effectiveness.
The technology has the ability to improve gene therapy for many hereditary diseases, especially inherited eye diseases. It has shown significant promise in correcting the vision impairment in animal models of blindness.
Prof Abhay Karandikar, Director of IIT Kanpur, said, “Gene therapy using viral vectors has recently emerged as a potent tool in the field of molecular medicine. We believe that this technology holds great promise for treating a wide range of hereditary eye diseases including Leber congenital amaurosis, an eye disorder that is present from birth; and Retinitis pigmentosa, a disease causing progressive sustained vision loss.”
Gene therapy is one of the most potent applications of recombinant DNA technology wherein pieces of DNA from multiple sources are combined to efficiently deliver the healthy copy of the faulty gene in a manner that production of protein from the introduced gene is sustained for life.
To enable this, the therapeutic DNA molecule is delivered using a virus that can infect human cells. For successful clinical application, several optimizations of the basic process is needed aimed at ensuring adequate expression of the desired gene.