Antisense technology gene therapy

Kleimnan et al. 2008). In addition, synthetic siRNAs are also subject to degradation in vivo by nuclease activity. Besides side effects and instability, the efficient and specific delivery of the RNAi indncers to the target cell still requires optimization. Here we snmmarize the cnrrent statns of nncleic acid-based antiviral therapentics. The focns will be on antiviral strategies nsing antisense and RNAi technology. Additionally, antiviral ribozymes and aptamers will be discussed briefly, with a focus on recent studies. Gene therapy approaches and delivery systems are the subject of Chapter 11 of this book. 

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. 

Despite the apparent success of antisense oligonucleotide therapy, several limitations of this technology have been manifested, First, the ODN must effectively cross the cell membrane to reach the cytoplasm or nucleus (permeation). Once inside the cell, the ODN must be resistant to degradation (stability). Finally the ODN must be able to bind specifically and with a high affinity to the RNA target to inhibit the desired gene (affinity and specificity) (28,30). 

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