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piggyBac-Based Non-Viral In Vivo Gene Delivery Useful for Production of Genetically Modified Animals and Organs

1
Section of Gene Expression Regulation, Frontier Science Research Center, Kagoshima University, Kagoshima 890-8544, Japan
2
Department of Pediatric Dentistry, Graduate School of Medical and Dental Sciences, Kagoshima University, Kagoshima 890-8544, Japan
3
Division of Pediatric Dentistry, Department of Oral Health Sciences, Course for Oral Life Science, Graduate School of Medical and Dental Sciences, Niigata University, Niigata 951-8514, Japan
4
Animal Genome Unit, Institute of Livestock and Grassland Science, National Agriculture and Food Research Organization (NARO), 2 Ikenodai, Tsukuba, Ibaraki 305-0901, Japan
5
Division of Biomedical Engineering, National Defense Medical College Research Institute, Saitama 359-8513, Japan
*
Author to whom correspondence should be addressed.
Pharmaceutics 2020, 12(3), 277; https://doi.org/10.3390/pharmaceutics12030277
Received: 7 February 2020 / Revised: 17 March 2020 / Accepted: 18 March 2020 / Published: 19 March 2020
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
In vivo gene delivery involves direct injection of nucleic acids (NAs) into tissues, organs, or tail-veins. It has been recognized as a useful tool for evaluating the function of a gene of interest (GOI), creating models for human disease and basic research targeting gene therapy. Cargo frequently used for gene delivery are largely divided into viral and non-viral vectors. Viral vectors have strong infectious activity and do not require the use of instruments or reagents helpful for gene delivery but bear immunological and tumorigenic problems. In contrast, non-viral vectors strictly require instruments (i.e., electroporator) or reagents (i.e., liposomes) for enhanced uptake of NAs by cells and are often accompanied by weak transfection activity, with less immunological and tumorigenic problems. Chromosomal integration of GOI-bearing transgenes would be ideal for achieving long-term expression of GOI. piggyBac (PB), one of three transposons (PB, Sleeping Beauty (SB), and Tol2) found thus far, has been used for efficient transfection of GOI in various mammalian cells in vitro and in vivo. In this review, we outline recent achievements of PB-based production of genetically modified animals and organs and will provide some experimental concepts using this system. View Full-Text
Keywords: piggyBac; transposon; non-viral gene delivery; electroporation; hydrodynamics; genetically modified animals; gene of interest; long-term gene expression; chromosomal integration piggyBac; transposon; non-viral gene delivery; electroporation; hydrodynamics; genetically modified animals; gene of interest; long-term gene expression; chromosomal integration
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MDPI and ACS Style

Sato, M.; Inada, E.; Saitoh, I.; Watanabe, S.; Nakamura, S. piggyBac-Based Non-Viral In Vivo Gene Delivery Useful for Production of Genetically Modified Animals and Organs. Pharmaceutics 2020, 12, 277.

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