I believe that one of the reasons why gene therapy is advancing slowly is that it is not easy getting an ideal vector to deliver therapeutic gene efficiently followed by optimal and durable expression of the gene product in the target tissue. For example, in September 1999, the perceptions of the use of adenoviral vectors for gene therapy was altered when a patient exposed through the hepatic artery to a high dose of adenoviral vector succumbed to the toxicity related to vector administration (St George, 2003).
Another possible reason for slow progression of gene therapy is the issue of safety. According to data updated to June 2012 and presented by The Journal of Gene Medicine, since the onset of the first gene therapy clinical trial in 1989, more than 1800 new clinical trials have been approved globally (Pezzoli, Chiesa, De Nardo & Candiani, 2012). However, none of the gene delivery products has been approved yet by the Food and Drug Administration (FDA) or by the European Medicines Agency (EMA) for commercial use on humans.
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Consequently, I believed that the future of gene therapy will rely on the development of a new group of more effective, application-specific multifunctional vectors whose properties should be tailored to the type of nucleic acid to be transmitted and the cells and tissues to be targeted.
I believe that one of the technological advance likely to be the most influential in the next decade is recombinant DNA. The recombinant DNA technology will help solve the major challenge of gene therapy research. It will assist in identification of an ideal vector. The recombinant DNA technology will help in finding an ideal vector for diseases such as HIV, which is currently a puzzle yet to be solved.
References
Pezzoli, D., Chiesa, R., De Nardo, L., & Candiani, G. (2012). We still have a long way to go to effectively deliver genes!. Journal Of Applied Biomaterials & Functional Materials , 10 (2), 82-91. doi:10.5301/JABFM.2012.9707
St George, J. A. (2003). Gene therapy progress and prospects: adenoviral vectors. Gene Therapy , 10 (14), 1135.
