A Cellular Model for Huntington's Disease
Applications
Diagnosis and treatment of Huntington's disease.
Technical Summary
Huntington's disease (HD) is a genetically inherited disease that leads to
progressive neurodegeneration, resulting in motor disturbances, loss of memory
and cognition, and psychiatric manifestations. The underlying molecular and
biochemical basis of HD are not fully understood, and consequently there is no
treatment effective in delaying or preventing the onset and progression of this
devastating disorder. HD cell models are useful tools in elucidating the
mechanistic basis of HD. However, most of the reported cell models have used
transient transfection in which the expression levels of transfected proteins
vary greatly and alter cell function and viability.
The present invention provides methods and compositions for the study,
diagnosis, and treatment of HD. Compositions comprise stably transfected cell
lines that can serve as cellular models of HD, an antin associated with HD, and
monoclonal antibodies specific for the antigen. The neuronal cell lines provided
are morphologically and phenotypically similar to and contain the same cellular
defects as cells derived from animal models of HD and/or HD patients.
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