Structural Features of Small Molecules Targeting the RNA Repeat Expansion That Causes Genetically Defined ALS/FTD

Genetically defined amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), collectively named c9ALS/FTD, are triggered by hexanucleotide GGGGCC repeat expansions [r(G(4)C(2))(exp)] within the C9orf 72 gene. In these diseases, neuronal loss occurs through an interplay of deleterious p...

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Pubblicato in:ACS Chem Biol
Autori principali: Ursu, Andrei, Wang, Kye Won, Bush, Jessica A, Choudhary, Shruti, Chen, Jonathan L, Baisden, Jared T, Zhang, Yong-Jie, Gendron, Tania F, Petrucelli, Leonard, Yildirim, Ilyas, Disney, Matthew D
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2020
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7788565/
https://ncbi.nlm.nih.gov/pubmed/33196168
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acschembio.0c00049
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