C9orf72 ALS/FTD dipeptide repeat protein levels are reduced by small molecules that inhibit PKA or enhance protein degradation
Licata, Nausicaa V, Cristofani, Riccardo, Salomonsson, Sally, Wilson, Katherine M, Kempthorne, Liam, Vaizoglu, Deniz, D’Agostino, Vito G, Pollini, Daniele, Loffredo, Rosa, Pancher, Michael, Adami, Valentina, Bellosta, Paola, Ratti, Antonia, Viero, Gabriella, Quattrone, Alessandro, Isaacs, Adrian M, Poletti, Angelo, Provenzani, Alessandro
Published in The EMBO journal (04.01.2022)
Published in The EMBO journal (04.01.2022)
Get full text
Journal Article
PolyGR and polyPR knock-in mice reveal a conserved neuroprotective extracellular matrix signature in C9orf72 ALS/FTD neurons
Milioto, Carmelo, Carcolé, Mireia, Giblin, Ashling, Coneys, Rachel, Attrebi, Olivia, Ahmed, Mhoriam, Harris, Samuel S, Lee, Byung Il, Yang, Mengke, Ellingford, Robert A, Nirujogi, Raja S, Biggs, Daniel, Salomonsson, Sally, Zanovello, Matteo, de Oliveira, Paula, Katona, Eszter, Glaria, Idoia, Mikheenko, Alla, Geary, Bethany, Udine, Evan, Vaizoglu, Deniz, Anoar, Sharifah, Jotangiya, Khrisha, Crowley, Gerard, Smeeth, Demelza M, Adams, Mirjam L, Niccoli, Teresa, Rademakers, Rosa, van Blitterswijk, Marka, Devoy, Anny, Hong, Soyon, Partridge, Linda, Coyne, Alyssa N, Fratta, Pietro, Alessi, Dario R, Davies, Ben, Busche, Marc Aurel, Greensmith, Linda, Fisher, Elizabeth M C, Isaacs, Adrian M
Published in Nature neuroscience (01.04.2024)
Published in Nature neuroscience (01.04.2024)
Get full text
Journal Article
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo
Kempthorne, Liam, Vaizoglu, Deniz, Cammack, Alexander J, Carcole, Mireia, Suklai, Pacharaporn, Muralidharan, Bhavana, Kroll, Francois, Moens, Thomas G, Yshii, Lidia, Verschoren, Stijn, Holbling, Benedikt V, Katona, Eszter, Mikheenko, Alla, Coneys, Rachel, De Oliveira, Paula, Yong-Jie, Zhang, Jensen, Karen, Daughrity, Lillian M, Mcgown, Alexander, Ramesh, Tennore M, Van Den Bosch, Ludo, Rahim, Ahad A, Petrucelli, Leonard, Rihel, Jason, Isaacs, Adrian M
Published in bioRxiv (28.01.2024)
Published in bioRxiv (28.01.2024)
Get full text
Paper