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Clinical Implications of Basic Research
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Volume 353:1410-1411 September 29, 2005 Number 13
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Targeting Specific Cell Types with Silencing RNA
Bryan R. Williams, Ph.D.

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RNA interference, which is mediated by small, double-stranded fragments of RNA called short interfering RNA (siRNA), is now a popular method to silence the expression of specific genes. It exploits the ability of the short, double-stranded fragment to abrogate the expression of the gene that shares its sequence and is useful not only as a basic research tool but also for drug-target validation. But the greatest hope for RNA interference lies in the potential use of siRNA oligonucleotides as drugs. The issue that has long bedeviled oligonucleotide-based therapies is how to deliver these agents to specific cellular or tissue targets. . . . [Full Text of this Article]


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From the Department of Cancer Biology, Lerner Research Institute, Cleveland Clinic Foundation, Cleveland.


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