Huntington disease is a neurodegenerative disorder caused by pathological expansion of the huntingtin ( HTT ) gene, and no disease-modifying therapies are currently available. Two new studies demonstrate different genetic approaches to targeting the toxic Htt transcript Htt1a in knockin mouse models of Huntington disease, which might inform future strategies to lower mutant HTT in humans.
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Nature Reviews Neurology published a clinical update in Neurology on 26 May 2026.
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