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HDAC INHIBITORS TO TREAT CHARCOT-MARIE-TOOTH DISEASE
专利权人:
Wim Robberecht;Ludo Van Den Bosch;Constantin Van Outryve d'Ydewalle
发明人:
Ludo Van Den Bosch,Constantin Van Outryve d'Ydewalle,Wim Robberecht,Constantin Van Outryve dYdewalle
申请号:
US13877648
公开号:
US20130227717A1
申请日:
2011.10.06
申请国别(地区):
US
年份:
2013
代理人:
摘要:
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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