Proteostatic imbalance and protein spreading in amyotrophic lateral sclerosis von Cicardi, Maria Elena, Marrone, Lara, Azzouz, Mimoun, Trotti, Davide

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C9orf72 expansion disrupts ATM-mediated chromosomal break repair von Walker, Callum, Herranz-Martin, Saul, Karyka, Evangelia, Liao, Chunyan, Lewis, Katherine, Elsayed, Waheba, Lukashchuk, Vera, Chiang, Shih-Chieh, Ray, Swagat, Mulcahy, Padraig J, Jurga, Mateusz, Tsagakis, Ioannis, Iannitti, Tommaso, Chandran, Jayanth, Coldicott, Ian, De Vos, Kurt J, Hassan, Mohamed K, Higginbottom, Adrian, Shaw, Pamela J, Hautbergue, Guillaume M, Azzouz, Mimoun, El-Khamisy, Sherif F

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LRP-1-mediated intracellular antibody delivery to the Central Nervous System von Tian, Xiaohe, Nyberg, Sophie, Sharp, Paul S., Madsen, Jeppe, Daneshpour, Nooshin, Armes, Steven P., Berwick, Jason, Azzouz, Mimoun, Shaw, Pamela, Abbott, N. Joan, Battaglia, Giuseppe

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Encapsulation of Biomacromolecules within Polymersomes by Electroporation von Wang, Linge, Chierico, Luca, Little, Daniel, Patikarnmonthon, Nisa, Yang, Zhou, Azzouz, Mimoun, Madsen, Jeppe, Armes, Steven P., Battaglia, Giuseppe

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Adaptor protein complex 4 deficiency: a paradigm of childhood-onset hereditary spastic paraplegia caused by defective protein trafficking von Behne, Robert, Teinert, Julian, Wimmer, Miriam, D’Amore, Angelica, Davies, Alexandra K, Scarrott, Joseph M, Eberhardt, Kathrin, Brechmann, Barbara, Chen, Ivy Pin-Fang, Buttermore, Elizabeth D, Barrett, Lee, Dwyer, Sean, Chen, Teresa, Hirst, Jennifer, Wiesener, Antje, Segal, Devorah, Martinuzzi, Andrea, Duarte, Sofia T, Bennett, James T, Bourinaris, Thomas, Houlden, Henry, Roubertie, Agathe, Santorelli, Filippo M, Robinson, Margaret, Azzouz, Mimoun, Lipton, Jonathan O, Borner, Georg H H, Sahin, Mustafa, Ebrahimi-Fakhari, Darius

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SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits von Hautbergue, Guillaume M., Castelli, Lydia M., Ferraiuolo, Laura, Sanchez-Martinez, Alvaro, Cooper-Knock, Johnathan, Higginbottom, Adrian, Lin, Ya-Hui, Bauer, Claudia S., Dodd, Jennifer E., Myszczynska, Monika A., Alam, Sarah M., Garneret, Pierre, Chandran, Jayanth S., Karyka, Evangelia, Stopford, Matthew J., Smith, Emma F., Kirby, Janine, Meyer, Kathrin, Kaspar, Brian K., Isaacs, Adrian M., El-Khamisy, Sherif F., De Vos, Kurt J., Ning, Ke, Azzouz, Mimoun, Whitworth, Alexander J., Shaw, Pamela J.

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Proteinopathies as Hallmarks of Impaired Gene Expression, Proteostasis and Mitochondrial Function in Amyotrophic Lateral Sclerosis von Benson, Bridget C., Shaw, Pamela J., Azzouz, Mimoun, Highley, J. Robin, Hautbergue, Guillaume M.

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Towards 3D Bioprinted Spinal Cord Organoids von Han, Yilin, King, Marianne, Tikhomirov, Evgenii, Barasa, Povilas, Souza, Cleide Dos Santos, Lindh, Jonas, Baltriukiene, Daiva, Ferraiuolo, Laura, Azzouz, Mimoun, Gullo, Maurizio R, Kozlova, Elena N

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Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9 von Hudák, Anett, Roach, Matthew, Pusztai, Dávid, Pettkó-Szandtner, Aladár, Letoha, Annamária, Szilák, László, Azzouz, Mimoun, Letoha, Tamás

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Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47 von Wiseman, Jessica P, Scarrott, Joseph M, Alves-Cruzeiro, João, Saffari, Afshin, Böger, Cedric, Karyka, Evangelia, Dawes, Emily, Davies, Alexandra K, Marchi, Paolo M, Graves, Emily, Fernandes, Fiona, Yang, Zih-Liang, Coldicott, Ian, Hirst, Jennifer, Webster, Christopher P, Highley, J Robin, Hackett, Neil, Angyal, Adrienn, Silva, Thushan de, Higginbottom, Adrian, Shaw, Pamela J, Ferraiuolo, Laura, Ebrahimi-Fakhari, Darius, Azzouz, Mimoun

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Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model von Ralph, G Scott, Azzouz, Mimoun, Radcliffe, Pippa A, Day, Denise M, Carthy, Janine M, Leroux, Marie A, Lee, Debbie C P, Wong, Liang-Fong, Bilsland, Lynsey G, Greensmith, Linda, Kingsman, Susan M, Mitrophanous, Kyriacos A, Mazarakis, Nicholas D

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AAV9‐mediated AIRE gene delivery clears circulating antibodies and tissue T‐cell infiltration in a mouse model of autoimmune polyglandular syndrome type‐1 von Almaghrabi, Sarah, Azzouz, Mimoun, Tazi Ahnini, Rachid

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C9ORF72 hexanucleotide repeat exerts toxicity in a stable, inducible motor neuronal cell model, which is rescued by partial depletion of Pten von Stopford, Matthew J, Higginbottom, Adrian, Hautbergue, Guillaume M, Cooper-Knock, Johnathan, Mulcahy, Padraig J, De Vos, Kurt J, Renton, Alan E, Pliner, Hannah, Calvo, Andrea, Chio, Adriano, Traynor, Bryan J, Azzouz, Mimoun, Heath, Paul R, Kirby, Janine, Shaw, Pamela J

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SRSF1-dependent inhibition of C9ORF72-repeat RNA nuclear export: genome-wide mechanisms for neuroprotection in amyotrophic lateral sclerosis von Castelli, Lydia M., Cutillo, Luisa, Souza, Cleide Dos Santos, Sanchez-Martinez, Alvaro, Granata, Ilaria, Lin, Ya-Hui, Myszczynska, Monika A., Heath, Paul R., Livesey, Matthew R., Ning, Ke, Azzouz, Mimoun, Shaw, Pamela J., Guarracino, Mario R., Whitworth, Alexander J., Ferraiuolo, Laura, Milo, Marta, Hautbergue, Guillaume M.

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Plastin 3 Promotes Motor Neuron Axonal Growth and Extends Survival in a Mouse Model of Spinal Muscular Atrophy von Alrafiah, Aziza, Karyka, Evangelia, Coldicott, Ian, Iremonger, Kayleigh, Lewis, Katherin E, Ning, Ke, Azzouz, Mimoun

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Optimised and rapid pre-clinical screening in the SOD1(G93A) transgenic mouse model of amyotrophic lateral sclerosis (ALS) von Mead, Richard J, Bennett, Ellen J, Kennerley, Aneurin J, Sharp, Paul, Sunyach, Claire, Kasher, Paul, Berwick, Jason, Pettmann, Brigitte, Battaglia, Guiseppe, Azzouz, Mimoun, Grierson, Andrew, Shaw, Pamela J

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Loss of TMEM106B exacerbates C9ALS/FTD DPR pathology by disrupting autophagosome maturation von Bauer, Claudia S, Webster, Christopher P, Shaw, Allan C, Kok, Jannigje R, Castelli, Lydia M, Lin, Ya-Hui, Smith, Emma F, Illanes-Álvarez, Francisco, Higginbottom, Adrian, Shaw, Pamela J, Azzouz, Mimoun, Ferraiuolo, Laura, Hautbergue, Guillaume M, Grierson, Andrew J, De Vos, Kurt J

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Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS von Iannitti, Tommaso, Scarrott, Joseph M., Likhite, Shibi, Coldicott, Ian R.P., Lewis, Katherine E., Heath, Paul R., Higginbottom, Adrian, Myszczynska, Monika A., Milo, Marta, Hautbergue, Guillaume M., Meyer, Kathrin, Kaspar, Brian K., Ferraiuolo, Laura, Shaw, Pamela J., Azzouz, Mimoun

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AAV9-mediated central nervous system-targeted gene delivery via cisterna magna route in mice von Lukashchuk, Vera, Lewis, Katherine E, Coldicott, Ian, Grierson, Andrew J, Azzouz, Mimoun

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The AIRE -230Y Polymorphism Affects AIRE Transcriptional Activity: Potential Influence on AIRE Function in the Thymus von Lovewell, Thomas R J, McDonagh, Andrew J, Messenger, Andrew G, Azzouz, Mimoun, Tazi-Ahnini, Rachid

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