GIGAXONIN FUSION PROTEIN AND METHODS FOR TREATING GIANT AXONAL NEUROPATHY

摘要

The present disclosure relates generally to fusion proteins including gigaxonin coupled to a cell penetrant peptide. These fusion proteins can be used to treat GAN in a subject in need thereof. Administration of the fusion proteins allows for control of at least one of Galectin-1 (GAL-1) levels and phosphorylated vimentin protein levels, thereby mediating aggregation of vimentin and the formation of vimentin-free zones in cells.