Gene Therapy
The treatment of genetic diseases has appeared a daunting challenge because there seemed little to be done if the immutable basic blueprints of the body have a serious imperfection (Beutler Pp). Even fifty years ago it was possible to greatly improve the quality of life and to actually "save the lives of patients with some such genetic diseases" (Beutler Pp). Success approaches included dietary manipulation as in phenylketonuria or galactosemia, surgery to correct various deformities, and often avoidance of inciting factors in the environment, "as in acute intermittent porphyria" (Beutler Pp). However, until the latter part of the twentieth century, the possibility of actually changing the faulty genetic blueprints was beyond the imagination of the realistic medical scientist (Beutler Pp).
Today it is certain that success in gene therapy will require the application of various technique that are tailored to the disease that is being approached (Beutler Pp). The majority of hereditary disorders will require approaches that "result in prolonged expression of the transgene, although in some storage diseases, particularly Gancher disease, transient expression...
The degree of control and the range of tissue expression will vary from application to application, thus, the design of appropriate gene therapy will require understanding not only of the molecular biology of the gene that is being transferred, but of the pathogenesis and clinical course of the disease that is being treated as well (Beutler Pp).
Gene therapy is the fusion of medicine and a variety of scientific fields, including cell biology, molecular biology, and immunology and is subject to all the rigorous standards and guidelines of each of these fields (Beutler Pp). Both viral and nonviral strategies for the delivery of exogenous genetic material…
