Naphthyridine tetramer as a bivalent ligand targeting DNA expanded GGGGCC sequences

摘要

Expansion of GGGGCC (G4C2) hexanucleotide repeat located in the gene C9ORF72 is the most common genetic cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) diseases.1 Development of effective small molecules targeting G4C2 repeats may prove a promising strategy for diseases diagnosis, regulation and/or treatment.2