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Review
. 2021 Jun 2;23(4):78.
doi: 10.1208/s12248-021-00608-7.

Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects

Hui Zu  1 , Danchen Gao  2
Affiliations
Review

Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects

Hui Zu et al. AAPS J. .

Abstract

Gene therapy has been experiencing a breakthrough in recent years, targeting various specific cell groups in numerous therapeutic areas. However, most recent clinical studies maintain the use of traditional viral vector systems, which are challenging to manufacture cost-effectively at a commercial scale. Non-viral vectors have been a fast-paced research topic in gene delivery, such as polymers, lipids, inorganic particles, and combinations of different types. Although non-viral vectors are low in their cytotoxicity, immunogenicity, and mutagenesis, attracting more and more researchers to explore the promising delivery system, they do not carry ideal characteristics and have faced critical challenges, including gene transfer efficiency, specificity, gene expression duration, and safety. This review covers the recent advancement in non-viral vectors research and formulation aspects, the challenges, and future perspectives.

Keywords: formulation; gene therapy; lipid; non-viral vector; polymer; transfection.

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Figures

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Fig. 1
Vehicle materials
Fig. 2
Fig. 2
Delivery mechanism for non-viral gene vectors
Fig. 3
Fig. 3
Effects of gene vector properties and formulation factors on non-viral gene delivery systems
See this image and copyright information in PMC

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