Mechanism-based rescue of Munc18-1 dysfunction in varied encephalopathies by chemical chaperones

Guiberson NGL, Pineda A, Abramov D, Kharel P, Carnazza KE, Wragg RT, Dittman JS, Burre J.

Nat Commun, 2018

Summary:

Explore how Maestro technology was used to explore heterozygous mutations in the neuronal protein Munc18-1, and to offer novel strategies for the treatment of varied encephalopathies caused by this mutation.

Keywords: neurodegeneration, Munc18-1, epilepsy, Parkinson's disease, Alzheimer's disease