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Gene therapy matures in the clinic

Nature Biotechnology volume 30, pages 588–593 (2012)Cite this article

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To the Editor:

Gene therapy has promised much and delivered little for patients over many years, but a range of clinical projects are now showing clear signs of efficacy, giving considerable reassurance that technology from this field will soon enter into mainstream medicine. The studies showing clinical promise cover several different diseases and use a variety of vectors, ranging from simple oligonucleotides to replicating lytic viruses. However, one crucial unifying feature is that they all focus on realistic and achievable objectives, particularly in terms of effective delivery of the therapeutic agent to target cells, but also using levels and durations of transgene expression commensurate with the desired treatment outcome. Careful selection of disease targets on the basis of the vector systems available is an essential prerequisite to success. Here we highlight some of the more notable recent advances and try to put them into the context of the underpinning technological improvements; details of selected recent clinical studies are summarized in Table 1. These and many other areas of scientific and clinical progress were recently updated at a collaborative congress held in Brighton, UK, between the European Society of Gene & Cell Therapy (ESGCT) and the British Society for Gene Therapy (BSGT)1 (http://www.esgct.eu/index.php/en/congress2/previous-congress/congres-2011).

Table 1 Some recent advances in clinical gene therapy
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Author information

Authors and Affiliations

  1. ESGCT; Department of Oncology, President, ESGCT-BSGT Collaborative Congress; General Secretary, University of Oxford, Oxford, UK.,

    Leonard W Seymour

  2. General Secretary, ESGCT,

    Leonard W Seymour

  3. President, BSGCT, Centre for Immunodeficiency, University College London Institute of Child Health, London, UK.,

    Adrian J Thrasher

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  1. Leonard W Seymour
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Competing interests

L.W.S. owns equity in PsiOxus Therapeutics, a biotech company developing an oncolytic group B adenovirus.

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Seymour, L., Thrasher, A. Gene therapy matures in the clinic. Nat Biotechnol 30, 588–593 (2012). https://doi.org/10.1038/nbt.2290

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