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Evaluating and enhancing ribozyme reaction efficiency in mammalian cells.

Publication ,  Journal Article
Jones, JT; Sullenger, BA
Published in: Nat Biotechnol
September 1997

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.

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Published In

Nat Biotechnol

DOI

ISSN

1087-0156

Publication Date

September 1997

Volume

15

Issue

9

Start / End Page

902 / 905

Location

United States

Related Subject Headings

  • Transfection
  • RNA, Catalytic
  • RNA
  • Promoter Regions, Genetic
  • Polymerase Chain Reaction
  • Plasmids
  • Mutation
  • Mammals
  • Genetic Therapy
  • DNA Repair
 

Citation

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Jones, J. T., & Sullenger, B. A. (1997). Evaluating and enhancing ribozyme reaction efficiency in mammalian cells. Nat Biotechnol, 15(9), 902–905. https://doi.org/10.1038/nbt0997-902
Jones, J. T., and B. A. Sullenger. “Evaluating and enhancing ribozyme reaction efficiency in mammalian cells.Nat Biotechnol 15, no. 9 (September 1997): 902–5. https://doi.org/10.1038/nbt0997-902.
Jones JT, Sullenger BA. Evaluating and enhancing ribozyme reaction efficiency in mammalian cells. Nat Biotechnol. 1997 Sep;15(9):902–5.
Jones, J. T., and B. A. Sullenger. “Evaluating and enhancing ribozyme reaction efficiency in mammalian cells.Nat Biotechnol, vol. 15, no. 9, Sept. 1997, pp. 902–05. Pubmed, doi:10.1038/nbt0997-902.
Jones JT, Sullenger BA. Evaluating and enhancing ribozyme reaction efficiency in mammalian cells. Nat Biotechnol. 1997 Sep;15(9):902–905.

Published In

Nat Biotechnol

DOI

ISSN

1087-0156

Publication Date

September 1997

Volume

15

Issue

9

Start / End Page

902 / 905

Location

United States

Related Subject Headings

  • Transfection
  • RNA, Catalytic
  • RNA
  • Promoter Regions, Genetic
  • Polymerase Chain Reaction
  • Plasmids
  • Mutation
  • Mammals
  • Genetic Therapy
  • DNA Repair