Vdac1 inhibition for the treatment of peripheral demyelination

Here the inventors show, using in vivo imaging and viral approaches, that calcium released by mitochondrial VDAC1 directly induces Schwann cell demyelination via MAPK and c-jun activation after sciatic nerve injury and more importantly in diabetic neuropathy. Moreover, reduction of mitochondrial calcium release by VDAC1 silence or/and drug blocking strongly reduces the number of demyelinating Schwann cell in vivo and improve nerve conduction and neuromuscular activity in diabetic and Charcot-Marie Tooth disease models. Accordingly the present invention relates to a method of treating a peripheral demyelinating disease in a subject in need thereof comprising administering to the subject a therapeutically effective amount of an inhibitor of VDAC1 activity or expression.

Keywords: Demyelinating neuropathies (CMT, ALS, Diabetic neuropathy,Tangier disease, Metachromatic leukodystrophy, Fabry'sdisease, hypothyroïd neuropathies, ... ), VDAC inhibitor - peptide
Patent Application number: EP15305756.7

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