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

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 u...

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Bibliographic Details
Published in:Pharmaceutics 2020-03, Vol.12 (3), p.277
Main Authors: Sato, Masahiro, Inada, Emi, Saitoh, Issei, Watanabe, Satoshi, Nakamura, Shingo
Format: Article
Language:English
Subjects:
Animals
chromosomal integration
Chromosomes
Deoxyribonucleic acid
DNA
electroporation
Gene expression
gene of interest
Gene therapy
Genetic engineering
genetically modified animals
Genomes
hydrodynamics
Liver
long-term gene expression
Mutagenesis
non-viral gene delivery
piggybac
Plasmids
Researchers
Review
transposon
Vectors (Biology)
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Summary: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.
ISSN:1999-4923
1999-4923
DOI:10.3390/pharmaceutics12030277