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Amyotrophic lateral sclerosis and gene therapy

Nature Clinical Practice Neurology volume 2, pages 462–463 (2006)Cite this article

With the identification of the genes and proteins involved in the etiology of neurodegenerative diseases, and the development of new viral tools for gene delivery, modulating the function of disease-associated genes as a therapy has become an increasingly realistic possibility. This approach is particularly applicable to amyotrophic lateral sclerosis (ALS), the dominant clinical manifestation of which is weakness that progresses to paralysis, with ensuing respiratory failure and death within 3–5 years of onset. A significant proportion of familial ALS cases are caused by a dominant mutation in the gene encoding superoxide dismutase (SOD1) that confers an aberrant toxic property on the protein; in principle, these cases could be amenable to treatment employing a gene-silencing strategy.

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Authors and Affiliations

  1. Department of Neurosciences,

    Timothy M Miller & Don W Cleveland

  2. Laboratory for Cell Biology, Ludwig Institute for Cancer Research,

    Timothy M Miller & Don W Cleveland

  3. the University of California, San Diego, La Jolla, CA, USA

    Timothy M Miller & Don W Cleveland

  4. Director of the Center for Neurologic Study and a Skaggs Clinical Scholar at Scripps Research Institute, La Jolla, CA, USA

    Richard A Smith

Authors
  1. Timothy M Miller
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  2. Richard A Smith
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  3. Don W Cleveland
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Corresponding author

Correspondence to Don W Cleveland.

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Competing interests

DW Cleveland and RA Smith have a consulting agreement with Isis Pharmaceutical Corporation. Along with ongoing compensation, they and their institutional employers could materially benefit if a therapeutic for amyotrophic lateral sclerosis results from their work. TM Miller declared he has no competing interests.

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Miller, T., Smith, R. & Cleveland, D. Amyotrophic lateral sclerosis and gene therapy. Nat Rev Neurol 2, 462–463 (2006). https://doi.org/10.1038/ncpneuro0270

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