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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5760849/
https://www.ncbi.nlm.nih.gov/pubmed/29113975
http://dx.doi.org/10.15252/emmm.201707850

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