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4 . 2014

Use of siRNA in cardiovascular diseases therapy

Abstract

RNA-inteferance (RNAi) is a widespread and effective mechanism of specific suppression of gene expression by small RNA molecules. Gene therapy based on RNAi is a promising approach for the treatment of various diseases. There are a lot of in vivo studies demonstrat- ing the effectiveness of this type of therapy. However no RNAi-based protocols are approved for clinical use. There are three basic RNAi-based principles for the gene therapy: cell treatment with siRNA for inhibition of target gene expression; overexpression of specific miRNA; and applying of miRNA inhibitors to suppress endogenous miRNAs. At the present time, the main challenge for RNAi-based technology is the target delivery of small RNAs into the cells. In this review we describe the mechanism of siRNA and miRNA action, different ways of their delivery and examples of RNAi- based gene therapy approaches in the context of cardio-vascular diseases.

Keywords:gene-directed therapy, RNA-interference, small interfering RNA (siRNAs), therapy of cardiovascular diseases

Clin. Experiment. Surg. Petrovsky J. – 2014. – N 4. – Р. 12–19.

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CHIEF EDITOR
CHIEF EDITOR
Sergey L. Dzemeshkevich
MD, Professor (Moscow, Russia)

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