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Novel siRNA delivery strategy: a new “strand” in CNS translational medicine?

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Abstract

RNA interference has been envisaged as a powerful tool for molecular and clinical investigation with a great potential for clinical applications. In recent years, increased understanding of cancer biology and stem cell biology has dramatically accelerated the development of technology for cell and gene therapy in these areas. This paper is a review of the most recent report of innovative use of siRNA to benefit several central nervous system diseases. Furthermore, a description is made of innovative strategies of delivery into the brain by means of viral and non-viral vectors with high potential for translation into clinical use. Problems are also highlighted that might hamper the transition from bench to bed, analyzing the lack of reliable preclinical models with predictive validity and the lack of effective delivery systems, which are able to overcome biological barriers and specifically reach the brain site of action.

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Acknowledgments

The T.P. lab is funded by supported by the EU 7th Framework Programme [FP2007-2013] under grant agreements no 223326 and 223524; the EXTRAPLAST IIT project; Epigenomics Flagship Project EPIGEN, MIUR-CNR; PNR-CNR Aging Program 2012-2014; and the CANESTRO project by Regione Toscana. The authors sincerely thank Dr. Paolo Cerioni and Dr. Helen Gallagher and Dr. Darren Finlay for their invaluable help in revising and editing the manuscript.

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Authors and Affiliations

  1. Institute of Clinical Physiology, CNR, Siena, Italy

    Lisa Gherardini

  2. Center for MicroBioRobotics @SSSA, Istituto Italiano di Tecnologia, Viale Rinaldo Piaggio 34, 56025, Pontedera, Italy

    Giuseppe Bardi

  3. Institute of Neuroscience, CNR, Via Moruzzi, 1 56124, Pisa, Italy

    Mariangela Gennaro & Tommaso Pizzorusso

  4. Department of Neuroscience, Psychology, Drug Research and Child Health NEUROFARBA, University of Florence, Florence, Italy

    Tommaso Pizzorusso

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  1. Lisa Gherardini
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  2. Giuseppe Bardi
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  3. Mariangela Gennaro
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  4. Tommaso Pizzorusso
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Correspondence to Tommaso Pizzorusso.

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Gherardini, L., Bardi, G., Gennaro, M. et al. Novel siRNA delivery strategy: a new “strand” in CNS translational medicine?. Cell. Mol. Life Sci. 71, 1–20 (2014). https://doi.org/10.1007/s00018-013-1310-8

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  • DOI: https://doi.org/10.1007/s00018-013-1310-8

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