Somatic Gene Therapy

The function of a vector is to control how much of the therapeutic product is produced and where in the body it is produced. Effective gene therapy requires that a therapeutic gene be introduced into cells of the body in a form which allows the gene (DNA) to be transcribed into mRNA, for the mRNA to be properly processed within the cell, for the mRNA to be translated into a protein, and for this protein to be properly modified, to reach the appropriate compartment, and to properly function.

This a achieved by constructing an expression vector

In its simplest form, gene therapy for inherited metabolic disease involves introducing a gene into a patient to express an enzyme which the patient lacks because of an inherited genetic defect. Examples of such therapy would be the replacement of phenylalanine hydroxylase in patients with phenylketonuria or the low-density lipoprotein (LDL) receptor in patients with familial hypercholesterolemia. This approach to gene therapy would be applicable to most autosomal recessive diseases and many X-linked diseases where one copy of the normal gene or

Inborn errors of metabolism, like most disease, have both genetic and environmental components. Current therapy for inborn errors of metabolism focuses on the diagnosis of a genetic defect before the onset of clinical symptoms, which allows changes to be made in the environment of the patient to prevent the progression of the pathological disease process. Dietary therapy and drug therapy are not directed at altering the genetic component of the inborn error of metabolism, but rather its environmental component.