Articles Tagged With: huntingtin
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Antisense Oligonucleotide Treatment of Huntington’s Disease: A Novel Potential Treatment
In this Phase I/IIa clinical trial, investigators administered antisense huntingtin oligonucleotides intrathecally to patients with early Huntington’s disease (HD), and demonstrated safety, tolerability, and dose-dependent reduction in CSF mutant huntingtin. This approach now is being tested for clinical efficacy in HD in a Phase III clinical trial.
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Untangling the Factors Governing Huntington’s Disease Progression
In a study of more than 3,000 subjects, CAG-repeat-dependent factors affecting age at onset also influenced rates of progression of cognitive, motor, and functional impairments, providing optimism that developing interventions, such as gene silencing therapies, could provide benefit.