HDAC INHIBITORS TO TREAT CHARCOT-MARIE-TOOTH DISEASE
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Aug 29, 2013
app pub date -
Oct 6, 2011
filing date -
Oct 8, 2010
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Abstract
The present application relates to diseases in the peripheral nervous system, particularly hereditary neuropathies, most particularly, Charcot-Marie-Tooth (CMT) disease. It is shown that this disease is associated with decreased acetylated tubulin levels, which can be overcome by inhibition of histone deacetylases (HDACs). Using HDAC inhibitors, it is shown herein that the symptoms of the CMT phenotype can be overcome both in vitro and in vivo. Also provided herein are two different mouse models of CMT disease.
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