HDAC inhibitors to treat charcot-marie-tooth disease

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.

Patent Publication Number: US9238028

Year filing: 2014

Year approval: 2016

Year publication: 2016

Status: Assigned

Technology domains: Pharmaceuticals, Other special machines

Validated for IOF-key: Yes

Attributed to: Associatie KULeuven