Strategy to assess the efficiency of U1 RNA-hammerhead ribozyme constructs using GFP-tagged targets.

Abstract

The application of ribozymes for gene therapy of autosomal dominant diseases has become popularized in recent years. Further this technology has widespread utility in the treatment of any disease, acquired or inherited, by inhibition of gene expression. The design of ribozymes is usually accomplished using computer assisted design programs, however they are not very useful in predicting the behavior of the ribozyme in the in vivo setting. To overcome this technical challenge, we developed a simple in vivo strategy to accurately assess the efficiency of ribozyme cleavage that significantly enhances the computer based design programs.