CNS-Specific Ribozyme Expression

Methods : a Companion to Methods in Enzymology
Y Prasad, J L Whitton

Abstract

Targeted genetic deletion is a powerful tool for analysis of gene function, but the standard approaches carry certain inescapable disadvantages. First, deletion is ubiquitous; tissue-specific knockout cannot be obtained. Second, temporal regulation of depletion is unattainable; the deleted functions are absent throughout the animal's development. As a consequence, during ontogeny, other gene products may be able to compensate, filling the functional gap. Furthermore bifunctional proteins exist that fulfill one role during development and another in the mature organism; deletion will remove the early function and, if this is lethal, the later function will remain undetected. Third, if genes utilize alternative splicing to control protein expression, it is difficult to target one spliced mRNA while leaving intact its related, but different, siblings. We review how these problems may be circumvented using ribozymes to diminish gene expression in a tissue-specific and temporally regulated manner and provide guidelines for the design and delivery of active ribozymes in vivo. Such methods may be particularly useful for analysis of genes involved in ontogeny and function of the central nervous system, in which individual genes may be ...Continue Reading

Citations

Nov 2, 2002·Proceedings of the National Academy of Sciences of the United States of America·Bindu NanduriKevin D Raney
Sep 28, 2018·The Journal of Biological Chemistry·Alicia K ByrdKevin D Raney
Jan 23, 1999·The Journal of Biological Chemistry·J J Arnold, C E Cameron
Jan 30, 2003·The Journal of Biological Chemistry·Steven S CarrollDavid B Olsen
Jan 16, 2015·The Journal of Biological Chemistry·Alicia K Byrd, Kevin D Raney
Jan 7, 2005·The Journal of Biological Chemistry·Alan J TackettKevin D Raney

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