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

: 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 accomplis...

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Bibliographic Details
Published in:Experimental dermatology 2003-10, Vol.12 (5), p.712-715
Main Authors: Cserhalmi-Friedman, Peter B., Djabali, Karima, Dietz, Harry C., Christiano, Angela M.
Format: Article
Language:English
Subjects:
Animals
Biological and medical sciences
efficacy
Flow Cytometry
General aspects
Genetic Therapy - methods
GFP
Green Fluorescent Proteins
in vitro
Luminescent Proteins - genetics
Medical sciences
Mice
Mice, Hairless
Molecular Biology - methods
Retroviridae - genetics
ribozyme
RNA, Catalytic - genetics
RNA, Small Nuclear - genetics
Software Design
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Summary:: 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.
ISSN:0906-6705
1600-0625
DOI:10.1034/j.1600-0625.2003.00081.x