Evaluating and enhancing ribozyme reaction efficiency in mammalian cells.

Journal Article (Journal Article)

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 that the ribozyme can convert up to 49% of a specific substrate RNA to product in the cellular environment and that the efficiency of this reaction is apparently a function of the ribozyme's ability to find and bind to the substrate RNA in the cell. These observations suggest that trans-splicing ribozymes may become useful reagents to repair a therapeutically significant fraction of mutant RNAs associated with a variety of genetic diseases.

Full Text

Duke Authors

Cited Authors

  • Jones, JT; Sullenger, BA

Published Date

  • September 1997

Published In

Volume / Issue

  • 15 / 9

Start / End Page

  • 902 - 905

PubMed ID

  • 9306408

International Standard Serial Number (ISSN)

  • 1087-0156

Digital Object Identifier (DOI)

  • 10.1038/nbt0997-902


  • eng

Conference Location

  • United States