Disease targets for gene therapy

TABLE 7.1-2. Disease Targets for Gene Therapy Monogenic Diseases... 

Only tissues giving rise to the disease pathology are targeted for gene therapy. 

Indications (1) pathogenesis of heart disease and targets for gene therapy... 

Gene therapy may be defined as the administration of exogenous DNA, in the form of intact gene(s) for therapeutic purposes. There are some a priori characteristics for diseases that are likely to be attractive targets for gene therapy, and a fundamental contrast between the gene therapy of protein replacement, in comparison with protein synthesis regulation. 

An important target for gene therapy is the mitochondrial genome, mutations of which may be involved in many rare diseases. Such mitochondiiopathies may be amenable to treat-ment by genetic modifications... 

In general, adenoviral vectors are known to infect the target cells effectively, but it is noteworthy to keep in mind that safety always comes first because some adverse side effects could be critical to the health of patients. Other types of viral vectors, such as adeno-associated virus, also are used for gene therapy in many diseases, even though more studies on safety, as well as efficacy, still remain until successful human clinical use can be expected. As demonstrated by clinical reports, fusion of knowledge on the molecular biology of viral vectors and the diseases to be treated holds promises for the future of medicine. 

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