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LIBRIS Formathandbok  (Information om MARC21)
FältnamnIndikatorerMetadata
00003990naa a2200661 4500
001oai:gup.ub.gu.se/260504
003SwePub
008240528s2018 | |||||||||||000 ||eng|
0247 a https://gup.ub.gu.se/publication/2605042 URI
0247 a https://doi.org/10.15252/emmm.2017078502 DOI
040 a (SwePub)gu
041 a eng
042 9 SwePub
072 7a ref2 swepub-contenttype
072 7a art2 swepub-publicationtype
1001 a Simone, Roberto4 aut
2451 0a G-quadruplex-binding small molecules ameliorate C9orf72 FTD/ALS pathology invitro and invivo.
264 c 2017-11-07
264 1b EMBO,c 2018
520 a 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 the repeat RNA forming foci that sequester RNA-binding proteins and through toxic dipeptide repeat proteins generated by repeat-associated non-ATG translation. GGGGCC repeat RNA folds into a G-quadruplex secondary structure, and we investigated whether targeting this structure is a potential therapeutic strategy. We performed a screen that identified three structurally related small molecules that specifically stabilise GGGGCC repeat G-quadruplex RNA We investigated their effect in C9orf72 patient iPSC-derived motor and cortical neurons and show that they significantly reduce RNA foci burden and the levels of dipeptide repeat proteins. Furthermore, they also reduce dipeptide repeat proteins and improve survival invivo, in GGGGCC repeat-expressing Drosophila Therefore, small molecules that target GGGGCC repeat G-quadruplexes can ameliorate the two key pathologies associated with C9orf72 FTD/ALS These data provide proof of principle thattargeting GGGGCC repeat G-quadruplexes has therapeutic potential.
650 7a MEDICIN OCH HÄLSOVETENSKAPx Medicinska och farmaceutiska grundvetenskaperx Neurovetenskaper0 (SwePub)301052 hsv//swe
650 7a MEDICAL AND HEALTH SCIENCESx Basic Medicinex Neurosciences0 (SwePub)301052 hsv//eng
7001 a Balendra, Rubika4 aut
7001 a Moens, Thomas G4 aut
7001 a Preza, Elisavet4 aut
7001 a Wilson, Katherine M4 aut
7001 a Woodling, Nathan S4 aut
7001 a Niccoli, Teresa4 aut
7001 a Gilbert-Jaramillo, Javier4 aut
7001 a Abdelkarim, Samir4 aut
7001 a Clayton, Emma L4 aut
7001 a Clarke, Mica4 aut
7001 a Konrad, Marie-Therese4 aut
7001 a Nicoll, Andrew J4 aut
7001 a Mitchell, Jamie S4 aut
7001 a Calvo, Andrea4 aut
7001 a Chio, Adriano4 aut
7001 a Houlden, Henry4 aut
7001 a Polke, James M4 aut
7001 a Ismail, Mohamed A4 aut
7001 a Stephens, Chad E4 aut
7001 a Vo, Tam4 aut
7001 a Farahat, Abdelbasset A4 aut
7001 a Wilson, W David4 aut
7001 a Boykin, David W4 aut
7001 a Zetterberg, Henrik,d 1973u Gothenburg University,Göteborgs universitet,Institutionen för neurovetenskap och fysiologi, sektionen för psykiatri och neurokemi,Institute of Neuroscience and Physiology, Department of Psychiatry and Neurochemistry4 aut0 (Swepub:gu)xzethe
7001 a Partridge, Linda4 aut
7001 a Wray, Selina4 aut
7001 a Parkinson, Gary4 aut
7001 a Neidle, Stephen4 aut
7001 a Patani, Rickie4 aut
7001 a Fratta, Pietro4 aut
7001 a Isaacs, Adrian M4 aut
7102 a Göteborgs universitetb Institutionen för neurovetenskap och fysiologi, sektionen för psykiatri och neurokemi4 org
7730 t EMBO molecular medicined : EMBOg 10:1, s. 22-31q 10:1<22-31x 1757-4684x 1757-4676
8564 u https://doi.org/10.15252/emmm.201707850
8564 8u https://gup.ub.gu.se/publication/260504
8564 8u https://doi.org/10.15252/emmm.201707850

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