Gene therapy nonviral delivery methods

Investigational gene therapies are comprised of two components the DNA itself (the construct ) and usually a method of delivery ( the vector ). Naked DNA can be injected but its expression is inefficient. Vectors may include viruses. However, such viruses have to be human, and their attenuation sometimes is lost after administration, leading to very serious adverse events. Nonviral vectors can include targeted liposomes, microspheres and emulsions. 

In this chapter, the delivery issues of DNA, especially plasmid DNA, are discussed with reference to gene therapy, focusing on existing biological barriers to be overcome and the characteristics of delivery methods and vectors to achieve this. Current progress and the status of nonviral gene delivery approaches are described. 

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