G‐quadruplex‐binding small molecules ameliorate C9orf72 FTD/ALS pathology in vitro and in vivo

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. Repeat expansions have been proposed to cause disease by both th...

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Detalhes bibliográficos
Publicado no:EMBO Mol Med
Main Authors: Simone, Roberto, Balendra, Rubika, Moens, Thomas G, Preza, Elisavet, Wilson, Katherine M, Heslegrave, Amanda, Woodling, Nathan S, Niccoli, Teresa, Gilbert‐Jaramillo, Javier, Abdelkarim, Samir, Clayton, Emma L, Clarke, Mica, Konrad, Marie‐Therese, Nicoll, Andrew J, Mitchell, Jamie S, Calvo, Andrea, Chio, Adriano, Houlden, Henry, Polke, James M, Ismail, Mohamed A, Stephens, Chad E, Vo, Tam, Farahat, Abdelbasset A, Wilson, W David, Boykin, David W, Zetterberg, Henrik, Partridge, Linda, Wray, Selina, Parkinson, Gary, Neidle, Stephen, Patani, Rickie, Fratta, Pietro, Isaacs, Adrian M
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2017
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Reports
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5760849/
https://ncbi.nlm.nih.gov/pubmed/29113975
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201707850
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