Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model (Neuron)

Nguyen et al. show that targeting polyGA proteins with a human antibody reduces GA aggregates and improves survival, behavior, and neurodegeneration in C9-BAC mice. These data demonstrate that RAN proteins drive disease and describe a novel therapeutic strategy for C9orf72 ALS/FTD.