Symposia.

In I C9orf72 i BAC transgenic ALS/FTD mice, inhibiting PKR through AAV expression of the dominant negative PKR-K296R protein decreases RAN protein pathology I in vivo i and improves behavioral phenotypes. These data demonstrate RAN proteins play a central role in I C9orf72 i ALS/FTD and describe novel approaches for the treatment of C9 and other RAN-protein diseases. Furthermore, alterations in protein SUMOylation are observed in a wide range of neurological and neurodegenerative diseases, and several extranuclear disease-associated proteins have been shown to be directly SUMOylated. Palmitoylation is a reversible post-translational modification that regulates membrane association, trafficking, and protein-protein interactions. Together these data suggest that a transmissible misfolded protein might play a role, and the prion protein could be directly or indirectly involved in the generation of the subtle MS-like pathology in recipient mice. [Extracted from the article]