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Clinical Implications of Basic Research
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Volume 348:1282-1283 March 27, 2003 Number 13
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Delivery on Demand — A New Era of Gene Therapy?
Josef T. Prchal, M.D.

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The discovery of the DNA code touched off an explosion of our knowledge of the molecular basis of human diseases and sparked ideas about treating inherited diseases with genes. Unfortunately, the first widely publicized attempt at gene therapy, in which the {beta}-globin gene was injected into a patient with {beta}-thalassemia, undertaken 20 years ago, failed because it was not based on an understanding of gene regulation. This ill-conceived experiment led to a public backlash and an unwarranted fear, bordering on hysteria, of recombinant-DNA research and its clinical applications. It soon became clear that identifying a gene sequence and a disease-causing . . . [Full Text of this Article]


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From the Department of Medicine, Baylor College of Medicine, Houston.


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