Delivery Systems in Gene Therapy
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Gene therapy includes the treatment of both genetically based and infectious diseases by introducinggenetic materials which have therapeutic effectsel-31. In its simplest terms, a wild type gene (which is non-functional in the cell leading to disease development) is introduced into the somatic cell lacking this gene torestore the normal gene function in this ceil. Many gene therapy strategies, however, utilize genes to destroyspecific cells. Such strategy is widely encountered in cancer gene therapy. Another gene therapy strategyis found in the diseases of the nervous system where the genetic basis is very complicated or not wellunderstood. Therapeutic genes in this case encode for a protein which is missing in the neuro-cells. The loss ofdopaminergic neurons, for example, plays a ma]or role in the development of Parkinson's disease.Therefore, genes that can enhance dopamine production will have therapeutic effects. Genes can also boostthe body's defense system against foreign infectious microorganisms. Gene therapy for humanimmunodeficiency virus (HIV), which relies on boosting T-cell immunity, for instance, has entered phase Iclinical trails.
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