Reversal of Neuropathology and Motor Dysfunction
in a Conditional Model of Huntington's Disease
Ai Yamamoto, Jose J.Lucas, and Rene Hen
Cell
101, 57-66 (2000)
Abstract:
Huntington's disease (HD) is an inherited (autosomal dominant) disorder
in which there is progressive neuro-degeneration, affecting the corpus
striatum and cerebral cortex of the brain, and for which there is no known
cure. Conditional transgenic mice model have been created by using tet-regulatable
system therefore could be a powerful means to investigate the relationship
of mutant protein and progression of the disease. Mice that expressing
a mutated huntingtin developed a neurodegenerative syndrome closely models
the human disease. Abolishing expression of the mutant protein not only
halts progression of the neuropathology but also reverse aggregate formation
and progressive motor decline. The results provide new targets for therapeutic
interventions for patients suffering from HD and suggest new therapeutic
approaches aimed either to destroy or inactivate the mutant huntingtin
protein might be effective.
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