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. Author manuscript; available in PMC: 2021 Oct 5.
Published in final edited form as: Chem Soc Rev. 2020 Oct 5;49(19):7167–7199. doi: 10.1039/d0cs00560f

Fig. 12.

Fig. 12

Small molecule targeting of r(G4C2)exp, the most common genetic cause of C9orf72-associated frontotemporal dementia and amyotrophic lateral sclerosis (c9FTD/ALS). (A) r(G4C2)exp sequesters hnRNP H, resulting in splicing defects. The repeat expansion also undergoes RAN translation and forms RNA foci. (B) Structures of compounds that bind to r(G4C2)exp. Compound 1a also binds to r(CGG)exp due to the 5′-CGG/3’-GGC binding site shared between the two repeats.