Opening the Floodgates: Clinically Applicable Hydrodynamic Delivery of Plasmid DNA to Skeletal Muscle

A number of issues hinder the translation of the concept of gene therapy into clinical practice. Of these, perhaps the most fundamental issue is the efficiency of gene delivery. Considerable effort has been and is being expended on developing the optimal vector and method of administration. The use of plasmid DNA as a vector system is an attractive prospect, as it is the simplest vector system to manufacture in the large quantities required for human treatment, its quality is relatively easy to control compared to complex viruses, and it can be stored for long periods of time in a lyophilized form.