4.8 Review

Are immunotherapies for Huntington's disease a realistic option?

期刊

MOLECULAR PSYCHIATRY
卷 24, 期 3, 页码 364-377

出版社

NATURE PUBLISHING GROUP
DOI: 10.1038/s41380-018-0021-9

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资金

  1. Fonds de Recherche du Quebec en Sante (FRQS) [35059]
  2. Canadian Institutes of Health Research (CIHR) [MOP326050]
  3. Universite Laval
  4. Fondation du CHU de Quebec

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There is compelling evidence that the pathophysiology of many neurodegenerative diseases includes dysregulation of the immune system, with some elements that precede disease onset. However, if these alterations are prominent, why have clinical trials targeting this system failed to translate into long-lasting meaningful benefits for patients? This review focuses on Huntington's disease, a genetic disorder marked by notable cerebral and peripheral inflammation. We summarize ongoing and completed clinical trials that have involved pharmacological approaches to inhibit various components of the immune system and their pre-clinical correlates. We then discuss new putative treatment strategies using more targeted immunotherapies such as vaccination and intrabodies and how these may offer new hope in the treatment of Huntington's disease as well as other neurodegenerative diseases.

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