Caister Academic Press

Therapeutic Potential of RNA-mediated Control of Gene Expression: Options and Designs

Lisa Scherer and John J. Rossi
from: RNA and the Regulation of Gene Expression: A Hidden Layer of Complexity (Edited by: Kevin V. Morris). Caister Academic Press, U.K. (2008)


We review factors to consider when choosing an mRNA knockdown approach in human cells using therapeutic expressed RNAs. We emphasize methods that use RNA triggers of endogenous cellular pathways to target and degrade mRNAs–RNAu, RNase P external guide sequences, tRNAse ZL small guide sequences, and RNAi– rather than those RNAs with intrinsic activity, such as ribozymes and aptamers. The range of available methods may be particularly important in combinatorial approaches to inhibit viruses prone to mutational escapes, such as HIV-1 and HCV read more ...
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