Unraveling the Molecular Basis for G‐Quadruplex‐Binders to ALS/FTD‐Associated G4C2 Repeats of the C9orf72 Gene
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DOI: 10.1002/cbic.202400974
Abstract: The most recurrent familial cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) is the presence of an abnormal number of intronic GGGGCC (G4C2) repetitions in the C9orf72 gene, which has been proposed to… read more here.
Keywords: molecular basis; c9orf72 gene; als ftd; g4c2 ... See more keywords
The Hairpin Form of r(G4C2)exp in c9ALS/FTD Is Repeat-Associated Non-ATG Translated and a Target for Bioactive Small Molecules.
Sign Up to like & getrecommendations! Published in 2019 at "Cell chemical biology"
DOI: 10.1016/j.chembiol.2018.10.018
Abstract: The most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) is an expanded G4C2 repeat [(G4C2)exp] in C9ORF72. ALS/FTD-associated toxicity has been traced to the RNA transcribed from the repeat expansion… read more here.
Keywords: associated non; g4c2 exp; g4c2; repeat associated ... See more keywords
Selective C9orf72 G-Quadruplex-Binding Small Molecules Ameliorate Pathological Signatures of ALS/FTD Models
Sign Up to like & getrecommendations! Published in 2022 at "Journal of Medicinal Chemistry"
DOI: 10.1021/acs.jmedchem.2c00654
Abstract: The G-quadruplex (G4) forming C9orf72 GGGGCC (G4C2) expanded hexanucleotide repeat (EHR) is the predominant genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Developing selective G4-binding ligands is challenging due to the conformational… read more here.
Keywords: quadruplex binding; binding small; c9orf72; g4c2 ... See more keywords
Crystal structure of parallel G-quadruplex formed by the two-repeat ALS- and FTD-related GGGGCC sequence
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DOI: 10.1093/nar/gkab302
Abstract: Abstract The hexanucleotide repeat expansion, GGGGCC (G4C2), within the first intron of the C9orf72 gene is known to be the most common genetic cause of both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The… read more here.
Keywords: crystal structure; g4c2; repeat; als ftd ... See more keywords
Crystal structures of distinct parallel and antiparallel DNA G-quadruplexes reveal structural polymorphism in C9orf72 G4C2 repeats
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DOI: 10.1093/nar/gkaf879
Abstract: Abstract The abnormal expansion of GGGGCC (G4C2) repeats in the noncoding region of the C9orf72 gene is a major genetic cause of two devastating neurodegenerative disorders, amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). These… read more here.
Keywords: c9orf72; crystal structures; g4c2; parallel antiparallel ... See more keywords
Supramolecular Polymorphism of (G4C2)n Repeats Associated with ALS and FTD
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DOI: 10.3390/ijms22094532
Abstract: Guanine-rich DNA sequences self-assemble into highly stable fourfold structures known as DNA-quadruplexes (or G-quadruplexes). G-quadruplexes have furthermore the tendency to associate into one-dimensional supramolecular aggregates termed G-wires. We studied the formation of G-wires in solutions… read more here.
Keywords: g4c2 formed; g4c2; g4c2 repeats; supramolecular polymorphism ... See more keywords