Gene Therapy — A Novel Form of Drug Delivery

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Abstract
Gene therapy is a novel form of drug delivery that enlists the synthetic machinery of the patient's cells to produce a therapeutic agent. Using the body to treat its own disease overcomes the need to manufacture highly purified proteins. It also eliminates the need for repeated parenteral administration of proteins (as in hemophilia) or drugs (as in hereditary hypercholesterolemia) and reduces the difficulties of complying with exogenous-drug regimens. Applications of gene therapy are not limited to rare inherited diseases but extend potentially to common acquired disorders, including cancer, heart disease, and the acquired immunodeficiency syndrome. Gene therapy is thus likely . . .

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