Hdac inhibitors to treat charcot-marie-tooth disease

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.

Patent Publication Number: EP2624832

Year filing: 2011

Year approval: 2017

Year publication: 2017

Status: Assigned

Technology domains: Pharmaceuticals, Other special machines

Validated for IOF-key: Yes

Attributed to: Associatie KULeuven