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The promise and potential hazards of adenovirus gene therapy
  1. L S YOUNG,
  1. CRC Institute for Cancer Studies
  2. University of Birmingham
  3. Birmingham B15 2TA, UK
  1. L S Young.L.S.Young{at}

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Gene therapy has been heralded as the “medicine of the 21st century”. With this has come an expectation which has yet to be met by experience. Thus the past decade has seen some 4000 patients entered into a variety of gene therapy trials with very few documented successes. Much of the “hype and hope” surrounding clinical gene therapy reflects the gap between the need to carefully perform phase I clinical trials to assess toxicity and tolerability and the public expectation that this approach is the panacea to chronic disease. Recent publicity highlights the current dilemma. The death in the USA of an 18 year old with ornithine transcarbamylase (OTC) deficiency after intrahepatic arterial injection of an adenovirus vector carrying a wild-type version of the defective enzyme has precipitated a flurry of reports and congressional hearings focusing on the ethics of such trials and on the very nature of clinical research itself.1 In contrast, a recent report provides one of the first clear cut gene therapy successes—the reconstitution of immune responses in two children with the SCID-X1 disorder by retroviral delivery of the wild-type gene to bone marrow.2 These two examples are interesting in that the therapies target rare monogenic diseases where returning the wild-type gene is the only rational therapeutic option while also posing the most rigorous test of the gene therapy approach—the ability to provide long term correction of gene defects. It is however in the area of the common ills such as heart disease and cancer that gene therapy promises so much and, despite current concerns, there is no doubt that with the necessary technical developments gene therapy will significantly improve the treatment of the major contemporary causes of premature mortality.

It is generally accepted that the major impediment to the successful application of gene therapy …

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