Treating the whole body in Huntington's disease

Jeffrey B. Carroll*, Gillian P. Bates, Joan Steffan, Carsten Saft, Sarah J. Tabrizi

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

120 Citations (Scopus)


Huntington's disease is a genetic neurodegenerative disorder with symptoms that are linked to the progressive dysfunction and neuronal death in corticostriatal circuits. The causative gene (mutated HTT) is widely expressed outside the CNS and several peripheral signs of disease, including weight loss and increased proinflammatory signalling, are often seen; however, their importance in the pathophysiology of Huntington's disease is not clear. Studies in animals have shown that features of the disease involving the CNS, including synapse loss and behavioural alterations, are susceptible to modulation by treatments that target tissues and organs outside the CNS. Links between peripheral biology and neurodegeneration have also been shown in other chronic neurodegenerative diseases, suggesting that modulation of these peripheral targets can offer new approaches to therapeutic development. Treatments targeted to tissues and organs outside the CNS might therefore substantially improve the quality of life of patients with Huntington's disease, even in the absence of disease-modifying effects.

Original languageEnglish
Article number147
Pages (from-to)1135-1142
Number of pages8
JournalLancet Neurology
Issue number11
Early online date11 Oct 2015
Publication statusPublished - 1 Nov 2015


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