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Int. J. Mol. Sci. 2018, 19(4), 1220;

Assembly and Functional Analysis of an S/MAR Based Episome with the Cystic Fibrosis Transmembrane Conductance Regulator Gene

Department of Biology and Biotechnology “Charles Darwin”, Sapienza University of Rome, 00185 Rome, Italy
Laboratory of Experimental and Regenerative Medicine, Department of Medical and Surgical Sciences, University of Foggia, 71122 Foggia, Italy
Department of Pediatrics and Infant Neuropsychiatry, Centro di Riferimento Fibrosi Cistica Regione Lazio, Sapienza University, 00165 Rome, Italy
Laboratory of Functional Biotechnology, Center of Sciences on Aging and Translational Medicine (CeSI-MeT), Department of Neuroscience, Imaging and Clinical Sciences, University G. d’Annunzio of Chieti-Pescara, 66013 Chieti, Italy
Authors to whom correspondence should be addressed.
Received: 24 February 2018 / Revised: 5 April 2018 / Accepted: 9 April 2018 / Published: 17 April 2018
(This article belongs to the Special Issue Gene Therapy)
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Improving the efficacy of gene therapy vectors is still an important goal toward the development of safe and efficient gene therapy treatments. S/MAR (scaffold/matrix attached region)-based vectors are maintained extra-chromosomally in numerous cell types, which is similar to viral-based vectors. Additionally, when established as an episome, they show a very high mitotic stability. In the present study we tested the idea that addition of an S/MAR element to a CFTR (cystic fibrosis transmembrane conductance regulator) expression vector, may allow the establishment of a CFTR episome in bronchial epithelial cells. Starting from the observation that the S/MAR vector pEPI-EGFP (enhanced green fluorescence protein) is maintained as an episome in human bronchial epithelial cells, we assembled the CFTR vector pBQ-S/MAR. This vector, transfected in bronchial epithelial cells with mutated CFTR, supported long term wt CFTR expression and activity, which in turn positively impacted on the assembly of tight junctions in polarized epithelial cells. Additionally, the recovery of intact pBQ-S/MAR, but not the parental vector lacking the S/MAR element, from transfected cells after extensive proliferation, strongly suggested that pBQ-S/MAR was established as an episome. These results add a new element, the S/MAR, that can be considered to improve the persistence and safety of gene therapy vectors for cystic fibrosis pulmonary disease. View Full-Text
Keywords: episome; S/MAR; cystic fibrosis; CFTR; gene therapy episome; S/MAR; cystic fibrosis; CFTR; gene therapy

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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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De Rocco, D.; Pompili, B.; Castellani, S.; Morini, E.; Cavinato, L.; Cimino, G.; Mariggiò, M.A.; Guarnieri, S.; Conese, M.; Del Porto, P.; Ascenzioni, F. Assembly and Functional Analysis of an S/MAR Based Episome with the Cystic Fibrosis Transmembrane Conductance Regulator Gene. Int. J. Mol. Sci. 2018, 19, 1220.

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