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

Peter B. Cserhalmi-Friedman, Karima Djabali, Harry C. Dietz, Angela M. Christiano

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
Pages (from-to)712-715
Number of pages4
JournalExperimental Dermatology
Volume12
Issue number5
DOIs
StatePublished - Oct 2003

Keywords

  • Efficacy
  • GFP
  • In vitro
  • Ribozyme

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Biology
  • Dermatology

Fingerprint Dive into the research topics of 'Strategy to assess the efficiency of U1 RNA-hammerhead ribozyme constructs using GFP-tagged targets'. Together they form a unique fingerprint.

Cite this