HDAC inhibitors to treat charcot-marie-tooth disease
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United States of America Patent
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Jan 19, 2016
Grant Date -
Nov 6, 2014
app pub date -
Jul 18, 2014
filing date -
Oct 8, 2010
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Abstract
The disclosure relates to diseases in the peripheral nervous system, particularly hereditary neuropathies, such as 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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