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Theodore Friedmann and Richard Roblin provided a brilliant prognosis of the potential and challenges of using gene therapy. Although this review highlighted the potential of this technique to treat disease, they also issued a warning to “oppose any future attempts at gene therapy in humans” until further knowledge is acquired and ethico-scientific criteria is agreed upon and followed.
In our view, gene therapy may ameliorate some human genetic diseases in the future. For this reason, we believe that research directed at the development of techniques for gene therapy should continue. For the foreseeable future, however, we oppose any further attempts at gene therapy in human patients because (i) our understanding of such basic processes as gene regulation and genetic recombination in human cells is inadequate; (ii) our understanding of the details of the relation between the molecular defect and the disease state is rudimentary for essentially all genetic diseases; and (iii) we have no information on the short-range and long-term side effects of gene therapy.T. Friedmann & R. Roblin