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The genie in the test tube from gene to gene therapy: part II

In the previous edition, we discussed gene therapy in terms of potential vectors and their applications. Now, we discuss the potential clinical uses of these constructs. To date, the main applications of gene therapy have been in inborn errors of metabolism, cancer, cardiovascular and central nervous system degenerative disease and transplantation and graft versus host disease. However, it would seem that this list is set to grow as understanding of the fundamental causes of disease and of delivery mechanisms for gene therapy become better understood.