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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