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Assays to Detect Small Molecules that Suppress Expression of the Mutant Huntington Disease Gene

ABSTRACTHuntington's disease (HD) is an autosomal dominant neurodegenerative disorder caused by a mutation in the huntingtin gene (HTT). JHU researchers approach to finding a treatment for HD is two-fold: 1) to find small molecules (that could be developed into clinically useful drugs) that can directly suppress expression of HTT by blocking activity...

Published: 3/13/2025 | Inventor(s): Russell Margolis, Dobrila Rudnicki, Wang Zheng, Pan Li, Xin Sun

Keywords(s): Assay, Clinical Diagnostics, CNS and Neurological Disorders, Disease Indication, Huntington's Disease, In Vitro Diagnostics, Neurodegeneration

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology > Huntington's Disease, Clinical and Disease Specializations > Neurology > Neurodegeneration, Clinical and Disease Specializations > Rare Diseases, Technology Classifications > Diagnostics > In Vitro Diagnostics, Technology Classifications > Research Tools > Assays, Technology Classifications > Diagnostics, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools