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NDT Advance Access originally published online on March 7, 2008
Nephrology Dialysis Transplantation 2008 23(7):2115-2118; doi:10.1093/ndt/gfn095
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© The Author [2008].
The online version of this article has been published under an open access model. Users are entitled to use, reproduce, disseminate, or display the open access version of this article for non-commercial purposes provided that: the original authorship is properly and fully attributed; the Journal and Oxford University Press are attributed as the original place of publication with the correct citation details given; if an article is subsequently reproduced or disseminated not in its entirety but only in part or as a derivative work this must be clearly indicated. For commercial re-use, please contact journals.permissions@oxfordjournals.org


Silencing genes in the kidney: antisense or RNA interference?

Jia-Hui Wang, Bruce M. Hendry and Claire C. Sharpe

Department of Renal Medicine, King's College London School of Medicine, London, UK

Correspondence and offprint requests to: Claire Sharpe, Department of Renal Medicine, King's College London School of Medicine, London, UK. Tel: +44-020-7848-5693/0491; Fax: +44-020-7848-0515; E-mail: claire.sharpe@kcl.ac.uk

Keywords: antisense; gene therapy; siRNA

The first 150 words of the full text of this article appear below.



   Introduction
 
Targeting genes in disease has long been a sought-after holy grail, with the concept of gene therapy promising a magic bullet for single gene mutation or deletion disorders. Replacing deficient or non-functional genes with the active form has proven more difficult to achieve than originally hoped. However, a subset of gene therapy termed ‘gene silencing’ is being developed, which is altogether more promising. By utilizing the unique nature of gene-sequence specificity, complementary or antisense molecules can be designed to ‘seek and destroy’ target mRNAs for specific proteins known to be pivotal in the pathogenesis of various disease processes. Two strategies have been employed to silence genes; the first involves the use of single-stranded antisense oligodeoxynucleotides (ASO) and the second uses double-stranded short-interfering RNA molecules (siRNA) otherwise known as RNA interference (RNAi).

Antisense oligodeoxynucleotides (ASO)
With the recent publication by Tillman et al. in the Journal of Pharmaceutical Sciences of the first demonstration . . . [Full Text of this Article]

Short-interfering RNA


   Conclusion
 

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