Research ArticleHuntington’s Disease

Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease

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Science Translational Medicine  03 Oct 2018:
Vol. 10, Issue 461, eaar3959
DOI: 10.1126/scitranslmed.aar3959

Rescuing cognition in Huntington’s disease

Huntington’s disease (HD) is a neurodegenerative disorder caused by mutation in the HTT gene. The encoded mutated protein, called huntingtin, acquires a toxic function causing motor, cognitive, and psychiatric impairments. Southwell and colleagues show that intracerebral injection of antisense oligonucleotides (ASOs) specifically inhibiting the expression of mutant Htt improved cognition and reduced anxiety and depressive behaviors in symptomatic HD mice. Moreover, HTT-targeting ASOs reduced huntingtin expression in nonhuman primates. The results suggest that ASO-based therapies might be effective for treating the cognitive impairments associated with HD.

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