It has been believed that nuclear gene delivery is the most important process for gene expression, and various non-viral vectors are currently being developed with this assumption. However, some of our earlier studies revealed a surprising difference in transfection activity between viral and non-viral vectors: this difference is largely due to the result of the intranuclear disposition of DNA ...

ment of acquired, refractory and fatal diseases in addition to inheritable gene deficiency diseases. Moreover, its application range is spreading to acute diseases or traumas. The gene delivery systems in vivo can be categorized as viral and nonviral approaches. Safety in usage of viral vectors for clinical gene therapy is not yet sufficient, whereas plasmid DNA (pDNA), which is a typical nonvi...

Sleeping Beauty (SB) is the first synthetic DNA transposon that was shown to be active in a wide variety of species. Here, we review studies from the last two decades addressing both basic biology and applications of this transposon. We discuss how host-transposon interaction modulates transposition at different steps of the transposition reaction. We also discuss how the transposon was transla...

The delivery of nucleic acids to the pulmonary route appears to be promising due to non-invasiveness, large area of epithelial surface lining the lung, easy accessibility and ability to provide a platform for local delivery. The therapeutic role of nucleic acids in various diseases pertaining to the pulmonary route e.g. cystic fibrosis, α1-antitrypsin deficiency, asthma, chronic obstructive pul...

Gene, short hairpin RNA (shRNA) and small interfering RNA (siRNA) delivery can be particularly used for the treatment of diseases by the entry of genetic materials mammalian cells either to express new proteins or to suppress the expression of proteins, respectively. Polyamidoamine (PAMAM) StarburstTM dendrimers are used as non-viral vectors (carriers) for gene, shRNA and siRNA delivery. Recent...

One of the major research focuses in the field of gene therapy is the development of clinically applicable, safe, and effective gene-delivery methods. Since the first case of human gene therapy was performed in 1990, a number of gene-delivery methods have been developed, evaluated for efficacy and safety, and modified for human application. To date, viral-vector-mediated deliveries have shown e...

While current antiretroviral therapy has significantly improved, challenges still remain in life-long targeting of HIV-1 reservoirs. Lentiviral gene therapy has the potential to deliver protective genes into the HIV-1 reservoir. However, inefficient reverse transcription (RT) occurs in HIV-1 reservoirs during lentiviral gene delivery. The viral protein Vpx is capable of increasing lentiviral RT...

A major obstacle in applying gene therapy to clinical practice is the lack of efficient and safe gene delivery techniques. Viral delivery has encountered a number of serious problems including immunological reactions and malignancy. Non-viral delivery methods (liposomes, sonoporation and electroporation) have either low efficiency invivo or produce severe collateral damage to ocular tissues. We...

Gene delivery vectors that are activated by external stimuli may allow improved control over the location and the degree of gene expression in target populations of cells. Light is an attractive stimulus because it does not cross-react with cellular signaling networks, has negligible toxicity, is noninvasive, and can be applied in space and time with unparalleled precision. We used the previous...

This chapter examines key concepts with respect to cancer gene therapy and the cur‐ rent issues with respect to non-viral delivery. The biological and molecular barriers that need to be overcome before effective non-viral delivery systems can be appropri‐ ately designed for oncology applications are highlighted and ways to overcome these are discussed. Strategies developed to evade the immune r...