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Formulation approaches to short interfering RNA and MicroRNA: Challenges and implications

Authors

  • Diana Guzman-Villanueva,

    1. Division of Pharmaceutics, College of Pharmacy, The University of Texas at Austin, Texas 78712-0120
    2. Facultad de Farmacia, Universidad Autonoma del Estado de Morelos, Cuernavaca 62209, Mexico
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  • Ibrahim M. El-Sherbiny,

    1. Division of Pharmaceutics, College of Pharmacy, The University of Texas at Austin, Texas 78712-0120
    2. Zewail City for Science and Technology, 6th October City, Giza, Egypt
    3. Chemistry Department, Faculty of Science, Mansoura University, Mansoura ET-35516, Egypt
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  • Dea Herrera-Ruiz,

    1. Facultad de Farmacia, Universidad Autonoma del Estado de Morelos, Cuernavaca 62209, Mexico
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  • Alexander V. Vlassov,

    1. Life Technologies, Austin, Texas 78744
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  • Hugh D. C. Smyth

    Corresponding author
    1. Division of Pharmaceutics, College of Pharmacy, The University of Texas at Austin, Texas 78712-0120
    • Division of Pharmaceutics, College of Pharmacy, The University of Texas at Austin, Texas 78712-0120. Telephone: +1 512-471-3383; Fax: +1 512-471-7474
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Abstract

RNA interference has emerged as a potentially powerful tool in the treatment of genetic and acquired diseases by delivering short interfering RNA (siRNA) or microRNA (miRNA) to target genes, resulting in their silencing. However, many physicochemical and biological barriers have to be overcome to obtain efficient in vivo delivery of siRNA and miRNA molecules to the organ/tissue of interest, thereby enabling their effective clinical therapy. This review discusses the challenges associated with the use of siRNA and miRNA and describes the nonviral delivery strategies used in overcoming these barriers. More specifically, emphasis has been placed on those technologies that have progressed to clinical trials for both local and systemic siRNA and miRNA delivery. © 2012 Wiley Periodicals, Inc. and the American Pharmacists Association J Pharm Sci 101:4046–4066, 2012

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