Mark W. Kankel

This study investigates the role of antisense and sense repeat RNAs in producing dipeptide repeat proteins in C9ORF72 expansion carriers and shows that targeting sense RNA with ASOs reduces both sense- and antisense-dependent DPR proteins.

This study evaluates human-derived anti-poly-GA antibodies in cellular and animal models of C9ORF72 disease, showing mixed effects on poly-GA levels and disease progression.

This study uses C. elegans models to show that eIF2D is essential for RAN translation in C9orf72 expansions, and its inhibition extends lifespan and reduces disease.