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Abstract
Introduction: Nucleic-acid-based biopharmaceuticals enclose a remarkable potential for treating debilitating or life-threatening diseases that currently remain incurable. This promising area of research envisages the creation of state-of-the-art DNA vaccines, pluripotent cells or gene-based therapies, which can be used to overcome current issues. To achieve this goal, DNA minicircles are emerging as ideal nonviral vectors due to their safety and persistent transgene expression in either quiescent or actively dividing cells.
Areas covered: This review focuses on the characteristics of minicircle DNA (mcDNA) technology and the current advances in their production. The possible modifications to further improve minicircle efficacy are also emphasized and discussed in light of recent advances. As a final point, the main therapeutic applications of mcDNA are summarized, with a special focus on pluripotent stem cells production and cancer therapy.
Expert opinion: Achieving in-target and persistent transgene expression is a challenging issue that is of critical importance for a successful therapeutic outcome. The use of miniaturized mcDNA cassettes with additional modifications that increase and prolong expression may contribute to an improved generation of biopharmaceuticals. The unique features of mcDNA render it an attractive alternative to overcome current technical issues and to bridge the significant gap that exists between basic research and clinical applications.
Declaration of interest
The authors disclose funding from Fundação para a Ciência e Tecnologia (FCT) which supports V Gaspar with an individual PhD fellowship (SFRH/BD/80402/2011). This work was also supported by the Technology FCT with the following projects (PEst-C/SAU/UI0709/2011 and PEst-OE/SAU/UI0709/2014). EC Costa acknowledges a fellowship (CENTRO-07-ST24-FEDER-002014) from “Programa Operacional Regional do Centro 2007-2013 QREN” (“Mais Centro” program). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Notes
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