Selective C9orf72 G-Quadruplex-Binding Small Molecules Ameliorate Pathological Signatures of ALS/FTD Models
The G-quadruplex (G4) forming C9orf72 GGGGCC (G4C2) expanded hexanucleotide repeat
(EHR) is the predominant genetic cause of amyotrophic lateral sclerosis (ALS) and …
(EHR) is the predominant genetic cause of amyotrophic lateral sclerosis (ALS) and …
G‐quadruplex‐binding small molecules ameliorate C9orf72 FTD/ALS pathology in vitro and in vivo
R Simone, R Balendra, TG Moens, E Preza… - EMBO molecular …, 2018 - embopress.org
Intronic GGGGCC repeat expansions in C9orf72 are the most common known cause of
frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), which are …
frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), which are …
Structural insight into C9orf72 hexanucleotide repeat expansions: Towards new therapeutic targets in FTD-ALS
Hexanucleotide repeat expansions,(G4C2) in the C9orf72 gene are considered as the
single most common genetic cause of both frontotemporal dementia (FTD) and amyotrophic …
single most common genetic cause of both frontotemporal dementia (FTD) and amyotrophic …
Characterizations of distinct parallel and antiparallel G-quadruplexes formed by two-repeat ALS and FTD related GGGGCC sequence
B Zhou, Y Geng, C Liu, H Miao, Y Ren, N Xu, X Shi… - Scientific reports, 2018 - nature.com
The large expansion of GGGGCC (G4C2) repeats of the C9orf72 gene have been found to
lead to the pathogenesis of devastating neurological diseases, amyotrophic lateral sclerosis …
lead to the pathogenesis of devastating neurological diseases, amyotrophic lateral sclerosis …
Three-dimensional structure of RNA monomeric G-quadruplex containing ALS and FTD related G4C2 repeat and its binding with TMPyP4 probed by homology …
K Mulholland, HJ Sullivan, J Garner, J Cai… - ACS chemical …, 2019 - ACS Publications
The G-quadruplex-forming hexanucleotide repeat expansion (HRE), d (G4C2) n, within the
human C9orf72 gene is the root cause for familial amyotrophic lateral sclerosis …
human C9orf72 gene is the root cause for familial amyotrophic lateral sclerosis …
Structural features of small molecules targeting the RNA repeat expansion that causes genetically defined ALS/FTD
A Ursu, KW Wang, JA Bush, S Choudhary… - ACS chemical …, 2020 - ACS Publications
Genetically defined amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD),
collectively named c9ALS/FTD, are triggered by hexanucleotide GGGGCC repeat …
collectively named c9ALS/FTD, are triggered by hexanucleotide GGGGCC repeat …
Advances in the structure of GGGGCC repeat RNA sequence and its interaction with small molecules and protein partners
X Liu, X Zhao, J He, S Wang, X Shen, Q Liu, S Wang - Molecules, 2023 - mdpi.com
The aberrant expansion of GGGGCC hexanucleotide repeats within the first intron of the
C9orf72 gene represent the predominant genetic etiology underlying amyotrophic lateral …
C9orf72 gene represent the predominant genetic etiology underlying amyotrophic lateral …
Topology of a G-quadruplex DNA formed by C9orf72 hexanucleotide repeats associated with ALS and FTD
B Zhou, C Liu, Y Geng, G Zhu - Scientific reports, 2015 - nature.com
Abnormal expansions of an intronic hexanucleotide GGGGCC (G4C2) repeat of the C9orf72
gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and …
gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and …
Enhanced insulin signalling ameliorates C9orf72 hexanucleotide repeat expansion toxicity in Drosophila
G4C2 repeat expansions within the C9orf72 gene are the most common genetic cause of
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The repeats …
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The repeats …
A small molecule exploits hidden structural features within the RNA repeat expansion that causes c9ALS/FTD and rescues pathological hallmarks
A Ursu, JT Baisden, JA Bush, A Taghavi… - ACS chemical …, 2021 - ACS Publications
The hexanucleotide repeat expansion GGGGCC [r (G4C2) exp] within intron 1 of C9orf72
causes genetically defined amyotrophic lateral sclerosis and frontotemporal dementia …
causes genetically defined amyotrophic lateral sclerosis and frontotemporal dementia …