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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
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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
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Technology Classifications > Diagnostics > In Vitro Diagnostics
,
Technology Classifications > Research Tools > Assays
,
Technology Classifications > Diagnostics
,
Clinical and Disease Specializations > Neurology
,
Technology Classifications > Research Tools
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