Advanced Concepts

These structures are not only useful in the laboratory, they are also potentially valuable in the clinic. Several structures have been proposed for use in antisense gene therapy Fig. 6-10. Introduction of sequences complementary to messenger RNA of a gene (antisense sequences) will prevent translation of that mRNA and expression of that gene. If this could be achieved in whole organisms, selected aberrantly expressed genes or even viral genes could be turned off. One drawback of this technology is the degradation of natural RNA and DNA by intracellu-lar nucleases. The nuclease-resistant structures are more stable and available to hybridize to the target mRNA.

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