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In gene therapy, DNA is inserted into human cells for therapeutic purposes. Although it was first contemplated as the treatment for monogenic diseases, there are other fields for its applications such as cancer and HIV. Over 1000 clinical trials have been carried out since 1990, and more than 5000 patients worldwide carry exogenous DNA in their cells. The efficiency and risks of gene therapy depend on the vehicle used for genetic material transfer. This review discusses the state of the art of gene therapy, the perspectives, and ethical issues for the implementation of this therapeutical alternative for inborn errors of metabolism.
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