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Sheetal K. Medhekar*, Namdeo G. Shinde, Riyaz Ali Osmani, Dhanashri U. Gadhave, Suyog P. Sulake, Shraddha M. Kumbhar 


Gene therapy is the technique in which defective or non-functional genes are replaced with targeted gene. Gene therapy has gained significant attention over the past two decades as a potential method for treating genetic disorders such as severe combined immunodeficiency, cystic fibrosis and Parkinson’s disease. Viral and on-viral vectors commonly used for gene delivery. Each of these vectors is designed to deliver normal copies of a gene into cells that contain only a mutated copy or defective gene. Viral vectors have been found to be dangerously toxic and this was tragically demonstrated when a 18 year old boy enrolled in gene therapy study had a massive immune reaction to the virus used resulting in death in only a few days due to multiple organ failure. This article chiefly focuses on the treatment of genetic disease by using activated polymer (dendrimers) and by use of siRNA technology. These approaches were made to deliver the drug to the targeted site and thus preventing toxicity and side effects to other cells and also reduce the chances of death in case of viral mediated gene transfer.

Keywords: Dendrimers, Gene therapy, Activated dendrimers, siRNA technology

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