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Gene Therapy Progress and Prospects: Episomally maintained self-replicating systems

Gene Therapy volume 11, pages 1735–1741 (2004)Cite this article

Abstract

The use of nonviral gene therapy vectors has been hampered by low level of transfection efficiency and lack of sustained gene expression. Episomal self-replicating systems may overcome these hurdles through their large packaging capacity, stability and reduced toxicity. This article reviews three classes of episomal molecules that have been tested with possible therapeutic genes: (1) self-replicating circular vectors, containing the Epstein–Barr virus (EBV) elements oriP and EBNA1; (2) small circular vectors containing scaffold/matrix attachment regions (S/MARs) as cis-acting elements to maintain the episomal status of the vector; (3) chromosomal vectors, based on the functional elements of the natural chromosomes. The studies reported validate the use of episomal vectors to obtain stable and prolonged gene expression, although reveal some limitations that necessitate additional work.

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Authors and Affiliations

  1. Institute for Experimental Treatment of Cystic Fibrosis, HS Raffaele, Milano, Italy

    M Conese

  2. c/o Dipartimento di Biologia Cellulare e dello Sviluppo, Istituto Pasteur-Fondazione Cenci Bolognetti, University of Rome ‘La Sapienza’, Rome, Italy

    C Auriche & F Ascenzioni

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  2. C Auriche
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Conese, M., Auriche, C. & Ascenzioni, F. Gene Therapy Progress and Prospects: Episomally maintained self-replicating systems. Gene Ther 11, 1735–1741 (2004). https://doi.org/10.1038/sj.gt.3302362

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