The objective for human gene therapy is to express exogenous DNA at the targeting cells or nearby to produce a practical and efficient therapeutic dosage at an appropriate time (quantitative pharmacology) with a safe manner. However, the use of genetic material as therapeutic agents has produced many novel and challenging obstacles that remain to be overcome. The obstacles include the formulation or packaging of the DNA, elements and construct of in vivo delivery system, penetration of biological barriers, DNA elimination within the cell and from the tissue compartments of the whole body, control of product expression and overt toxicity. Furthermore, the dose response and control of viral vectors can also be affected by viral serotypes, tissue tropisms, cell targeting, drug regulation, injection route, age and sex, etc. This review discusses the quantitative pharmacological control and effect of current viral vector based preclinical and clinical therapy.
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