Evaluating and enhancing ribozyme reaction efficiency in mammalian cells

  title={Evaluating and enhancing ribozyme reaction efficiency in mammalian cells},
  author={Joshua T. Jones and Bruce A. Sullenger},
  journal={Nature Biotechnology},
The ability of ribozymes to cleave specific transcripts and repair defective RNAs in the test tube has engendered speculation about their potential clinical utility. Therapeutic development has been hindered by an inability to evaluate and optimize the efficiency of RNA catalysis in vivo. We describe an experimental system that has allowed us to assess and enhance the efficiency with which a trans-splicing group I ribozyme reacts with a targeted RNA in mammalian cells. These results demonstrate… CONTINUE READING

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