Abstract
A reliable and specific modulation of gene expression in a cell would greatly facilitate the study of unknown gene functions, to combat pathogenic gene products or to control processes in biotechnology. One exciting option to achieve this goal is the application of antisense molecules such as antisense RNAs, antisense oligonucleotides or catalytic RNAs named ribozymes. All of these molecules bind to their target RNAs via complementary sequences, thereby forming base-paired duplices. Thus, in theory they permit a sequence-specific inhibition of gene expression. It is therefore not surprising that antisense approaches are increasingly used to understand the function of a given gene product, or to interfere with virus replication or cancerogenesis. Significant progress has been made in the design of antisense molecules, their delivery to cells, and in the understanding of their mode of action. However, compared to in vitro conditions, inside the cell the situation is much more complicated because of additional factors such as RNA-binding proteins and several intracellular compartments. Thus, in spite of the apparent simplicity of antisense approaches, many problems such as intracellular stability of the antisense molecule, target site selection, or colocalization of antisense molecule and target RNA remain to be solved. This review will address several of these problems and will discuss possible solutions. Due to space limitations, it is restricted to aspect of ribozyme applications.
Published Version
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