Ribonuclease Recruitment Using a Small Molecule Reduced c9ALS/FTD r(G4C2) Repeat Expansion In Vitro and In Vivo ALS Models

Scientists demonstrated that targeted small-molecule degradation of an expanded G4C2 RNA repeat [r(G4C2)exp] was a strategy for mitigating amyotrophic lateral sclerosis and frontotemporal dementia (c9ALS/FTD)-associated pathologies and studying disease-associated pathways in preclinical models.