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Selective C9orf72 G-Quadruplex-Binding Small Molecules Ameliorate Pathological Signatures of ALS/FTD Models

[Image: see text] 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 polym...

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Detalles Bibliográficos
Autores principales: Cheng, Aifang, Liu, Changdong, Ye, Wenkang, Huang, Duli, She, Weiyi, Liu, Xin, Fung, Chun Po, Xu, Naining, Suen, Monica Ching, Ye, Wei, Sung, Herman Ho Yung, Williams, Ian Duncan, Zhu, Guang, Qian, Pei-Yuan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Chemical Society 2022
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9574859/
https://www.ncbi.nlm.nih.gov/pubmed/36226410
http://dx.doi.org/10.1021/acs.jmedchem.2c00654