Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Impact of DNA Vector Topology on Non-Viral Gene Therapeutic Safety and Efficacy

Author(s): Chi H. Sum, Shawn Wettig, Roderick A. Slavcev.

Abstract:

Gene therapy continues to grow as an emerging treatment strategy toward numerous diseases. However, such prospects are hindered by the use of viral vectors prompting significant safety concerns along with limitations concerning repeat administrations, size of delivered gene construct, scale-up, high production costs, contamination during production, and lack of desired tissue selectivity. Non-viral gene delivery demonstrates the potential to address the abovementioned limitations, but itself generally suffers from low efficacy. Continuing efforts have been made to develop innovative delivery systems, synthetic gene carriers, and DNA vectors in a concerted attempt to enhance gene delivery suitable for clinical applications. In this review, we focus on the advances in the design of novel DNA vectors catered to enhance transfection and transgene expression and their influences on the efficacy and safety of existing and emerging delivery systems and synthetic vectors for non viral gene delivery.

Keywords: Cationic lipids, gene therapy, linear covalently closed (LCC) DNA minivector, lipoplex, non-viral gene delivery, recombinant plasmid DNA vectors, synthetic vectors.

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Article Details

VOLUME: 14
ISSUE: 4
Year: 2014
Page: [309 - 329]
Pages: 21
DOI: 10.2174/1566523214666140612154929