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Showing 1–3 of 3 results
Advanced filters: Author: Henry C Groff Clear advanced filters

  • Last year's Nobel Prize in Physiology or Medicine was won by Andrew Fire and Craig Mello for their discovery of RNA interference, an evolutionarily conserved cellular pathway that regulates endogenous gene expression. In this Review, Gonzalez and Paulson discuss how RNA interference is being exploited experimentally to suppress the expression of dominantly acting genes that cause incurable neurodegenerative disorders, and highlight the questions that need to be answered before human clinical trials of this technology can commence.

    • Pedro Gonzalez-Alegre
    • Henry L Paulson
    Reviews
    Nature Clinical Practice Neurology
    Volume: 3, P: 394-404

  • Several of the spinocerebellar ataxias (SCAs) result from expansion of polyglutamine (polyQ)-encoding regions in different genes. Here, Orr and colleagues examine the clinical features of the the polyQ SCAs, and suggest that understanding the molecular and physiological mechanisms underlying polyQ SCAs can inform therapeutic strategies for these and other polyQ disorders.

    • Henry L. Paulson
    • Vikram G. Shakkottai
    • Harry T. Orr
    Reviews
    Nature Reviews Neuroscience
    Volume: 18, P: 613-626