Antisense technology

In addition to some technical difficulties outlined earlier, a number of non-technical issues must be satisfactorily addressed before its practice becomes widespread. Chief amongst these issues are the questions of public perception, ethics and costs. 

Gene therapy is not, and will not be, an inexpensive therapeutic tool. The cost of such treatments will likely be broadly similar to the cost of present-day biopharmaceuticals. However, if proven successful in treating many currently incurable conditions, the cost beneht ratio will almost certainly greatly favour its medical use. 

Various disease states are associated with the inappropriate production/overproduction of gene products. Examples include  

An additional example includes the intracellular transcription and translation of virally encoded genes during intracellular viral replication. In all such instances, the medical consequences of such inappropriate gene (over)expression could be ameliorated/prevented if this expression could be down-regulated. A nucleic-acid-based approach to achieve just this is termed antisense technology . 

CH14 NUCLEIC-ACID- AND CELL-BASED THERAPEUTICS 

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The number of clinical trials ongoing represents a growing interest in antisense technology. 

Zamecnik P.C, Stephensen M.L., Proc. Natl Acad. Sci. USA 1978, 75, 280-284. Antisense technology Part A General methods, methods of delivery and RNA studies. In Phillips M.I. (Ed.), Methods in Enzymology Vol. 313. Academic Press San Diego, CA, 2000. 

Antisense technology Part B Applications. In Phillips M.I. (Ed.), Methods Enzymology Vol. 314. Academic Press ... 

Volume 313. Antisense Technology (Part A General Methods, Methods of Delivery, and RNA Studies)... 

Phillips, M. 2000. Antisense Technology. Methods in Enzymology, volume 313. Academic Press. Crooke, S. (Ed.) 2001. Antisense Drug Technology. Marcel Dekker. 

Kurreck, J. Antisense Technologies Improvement through Novel Chemical Modifications. European Journal of Biochemistry 270 no. 8 (2003) 1628-1644. 

Yet additional therapeutic approaches to AIDS, based upon antisense technology, will be discussed later in this chapter. The use of gene therapy to combat this disease has now become firmly established as an approach worthy of significant future research. 

Every few decades, a medical innovation is perfected that profoundly influences the practice of medicine. Widespread vaccination against common infectious agents and the discovery of antibiotics serve as two such examples. Many scientists now believe that the potential of gene therapy and antisense technology rivals even the most significant medical advances achieved to date. 

Wagner, R. Flanagan, W. (1997). Antisense technology and prospects for therapy of viral infections and cancer. Mol. Med. Today 3(1), 31-38. 

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