Cover Credit:Diseased upper motor neurons (UMNs) shrink in size and their apical dendrites display profound disintegration with numerous vacuoles. This phenomenon is observed both in UMNs of patients and UMNs of well-characterized disease models, regardless of species.
Upon UCHL1 gene therapy to diseased UMNs, they regain their neuronal stability, improve the integrity of their apical dendrite and soma.These results not only show that they respond to gene therapy, but also that directed gene therapy to UMNs would help restore UMN degeneration, especially in diseases that are characterized by UMN loss. See paper by Barış Genç et al. in this issue of Gene Therapy.