Fly model used in drug discovery study related to FTD and ALS (research report now available at PubMed Central)

Simone R, et al. G-quadruplex-binding small molecules ameliorate C9orf72 FTD/ALS pathology in vitro and in vivo. EMBO Mol Med. 2018 Jan;10(1):22-31. doi: 10.15252/emmm.201707850. PubMed PMID: 29113975; PubMed Central PMCID: PMC5760849.

From the abstract: "Intronic GGGGCC repeat expansions in C9orf72 are the most common known cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), which are characterised by degeneration of cortical and motor neurons, respectively. ... We performed a screen that identified three structurally related small molecules that specifically stabilise GGGGCC repeat G-quadruplex RNA ... Furthermore, they also reduce dipeptide repeat proteins and improve survival in vivo, in GGGGCC repeat-expressing Drosophila ... These data provide proof of principle that targeting GGGGCC repeat G-quadruplexes has therapeutic potential."