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siRNA-based therapeutic approaches for rheumatic diseases

Nature Reviews Rheumatology volume 9, pages 56–62 (2013)Cite this article

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Abstract

RNA interference (RNAi) is one of the most exciting and important discoveries of the past few decades. Small interfering RNAs (siRNAs) can silence gene activity and be used to interfere with pathophysiological processes. Substantial research has focused on introducing 'drug-like' properties—stability, selectivity and potency—to RNAi molecules, and clinical trials have been initiated. Despite initial success, the current challenge that remains is to develop optimized vehicles that avoid off-target effects whilst efficiently delivering the therapeutic siRNA to specific cell types. As for many other diseases, siRNA-based therapy is emerging as a promising approach for the treatment of rheumatic disorders. Although the pathogenesis of rheumatic diseases is complex, identification of candidate genes able to influence either inflammation or structural damage has been successful. Here, we will discuss advances in the field and potential applications of siRNA therapeutics in clinical trials for rheumatic conditions.

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Figure 1: Strategies that interfere with endogenous miRNA biogenesis for in vivo siRNA-based therapy.
Figure 2: Ideal strategy for optimal siRNA-based treatment in rheumatic diseases.

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Acknowledgements

The authors of this article are supported by grants from INSERM and the IMI EU funded project BeTheCure (contract n° 115142-2).

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  1. INSERM, U844, University Hospital Montpellier, 80 avenue Augustin Fliche, Montpellier, 34295, France

    Florence Apparailly & Christian Jorgensen

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  1. Florence Apparailly
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  2. Christian Jorgensen
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Both authors contributed equally to all aspects of this manuscript.

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Correspondence to Florence Apparailly.

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Apparailly, F., Jorgensen, C. siRNA-based therapeutic approaches for rheumatic diseases. Nat Rev Rheumatol 9, 56–62 (2013). https://doi.org/10.1038/nrrheum.2012.176

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