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      Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy

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          Abstract

          Purpose:

          Gene-mediated enzyme replacement is a reasonable and highly promising approach for the treatment of Fabry disease (FD). The objective of the present study was to demonstrate the potential applications of solid lipid nanoparticle (SLN)-based nonviral vectors for the treatment of FD.

          Methods:

          SLNs containing the pR-M10-αGal A plasmid that encodes the α-Galactosidase A (α-Gal A) enzyme were prepared and their in vitro transfection efficacy was studied in Hep G2 cells. We also studied the cellular uptake of the vectors and the intracellular disposition of the plasmid.

          Results:

          The enzymatic activity of the cells treated with the vectors increased significantly relative to the untreated cells, regardless of the formulation assayed. When the SLNs were prepared with protamine or dextran and protamine, the activity of the α-Gal A enzyme by the transfected Hep G2 cells increased up to 12-fold compared to that of untreated cells.

          Conclusion:

          With this work we have revealed in Hep G2 cells the ability of a multicomponent system based on SLNs to act as efficient nonviral vectors to potentially correct low α-Gal A activity levels in FD with gene therapy.

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          Author and article information

          Journal
          Drug Des Devel Ther
          Drug Des Devel Ther
          Drug Design, Development and Therapy
          Dove Medical Press
          1177-8881
          2012
          26 October 2012
          : 6
          : 303-310
          Affiliations
          Pharmacokinetics, Nanotechnology and Gene Therapy Group, Pharmacy Faculty, University of the Basque Country UPV/EHU, Vitoria-Gasteiz, Spain
          Author notes
          Correspondence: Alicia Rodríguez Gascón, Pharmacy and Pharmaceutical Technology Laboratory, Pharmacy Faculty, University of the Basque Country (UPV/EHU), Paseo de la Universidad 7, 01006 Vitoria-Gasteiz, Spain, Tel +34 945 013 094, Email alicia.rodriguez@ 123456ehu.es
          Article
          dddt-6-303
          10.2147/DDDT.S36131
          3484727
          23118528
          © 2012 Ruiz de Garibay et al, publisher and licensee Dove Medical Press Ltd.

          This is an Open Access article which permits unrestricted noncommercial use, provided the original work is properly cited.

          Categories
          Original Research

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