About: Antisense technology presents an opportunity to manipulate gene expression within the cells to treat various diseases, and acts as a powerful tool for studying gene function utilizing antisense agents to manage the diseases by regulating the expression of the specific factor that actually causes the particular disease. Highly specific and effective gene silencing of any disease can be achieved by an accurate knowledge of the target mRNA sequence and rational design of its complementary antisense agents for the downregulation of its protein message. This technology has been successfully used for the treatment of cancer, HIV, and other mutating viral diseases. The technology uses agents like antisense oligonucleotides, ribozymes, short interfering RNA (siRNA), microRNA (miRNA), apatamers, and others. The purpose of this chapter is to survey different antisense technologies and the various mechanisms of gene silencing. The chapter also highlights therapeutic limitations, benefits, clinical applications, and various advances to meet delivery challenges of these agents, and gives due consideration to the regulatory and patent aspects.   Goto Sponge  NotDistinct  Permalink

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  • Antisense technology presents an opportunity to manipulate gene expression within the cells to treat various diseases, and acts as a powerful tool for studying gene function utilizing antisense agents to manage the diseases by regulating the expression of the specific factor that actually causes the particular disease. Highly specific and effective gene silencing of any disease can be achieved by an accurate knowledge of the target mRNA sequence and rational design of its complementary antisense agents for the downregulation of its protein message. This technology has been successfully used for the treatment of cancer, HIV, and other mutating viral diseases. The technology uses agents like antisense oligonucleotides, ribozymes, short interfering RNA (siRNA), microRNA (miRNA), apatamers, and others. The purpose of this chapter is to survey different antisense technologies and the various mechanisms of gene silencing. The chapter also highlights therapeutic limitations, benefits, clinical applications, and various advances to meet delivery challenges of these agents, and gives due consideration to the regulatory and patent aspects.
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