Abstract
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.
Highlights
Viral vectors, including adenoviral (AV), adeno-associated viral (AAV), and lentiviral (LV) vectors, are most commonly used for gene delivery experiments in basic research and clinical gene therapy
A single dose of the hybrid vector led to life-long restoration of bile composition, prevention of biliary cirrhosis, and a substantial reduction in tumorigenesis. They concluded that this hybrid recombinant AAV (rAAV)/PB transposon vector strategy is powerful for correcting juvenile-onset chronic liver disease and reducing the tumorigenicity of Progressive familial intrahepatic cholestasis type 3 (PFIC3)
Tg animals have been frequently used for overexpression of gene of interest (GOI) in a specific tissue or cell type in vivo, but their production and propagation are time-consuming and costly
Summary
Viral vectors, including adenoviral (AV), adeno-associated viral (AAV), and lentiviral (LV) vectors, are most commonly used for gene delivery experiments in basic research and clinical gene therapy. Transposons ( called movable genetic elements) are DNA sequences that can move to different locations within a genome and are recognized as useful tools for non-viral gene delivery into mammalian cells [8] This gene delivery can be achieved by transporting a transposon carrying a gene of interest (GOI) and a transposase that mediates chromosomal integration of the GOI into cells. Examples of the diverse roles of the PB-based gene delivery system in the production of genetically modified (GM) animals and organs, along with some ideas on possible in vivo PB-based transfection of somatic cells and those useful for increasing PB transposition activity achieved far, will be mentioned
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