Proteostatic imbalance and protein spreading in amyotrophic lateral sclerosis
Cicardi, Maria Elena, Marrone, Lara, Azzouz, Mimoun, Trotti, Davide
Published in The EMBO journal (17.05.2021)
Published in The EMBO journal (17.05.2021)
Get full text
Journal Article
C9orf72 expansion disrupts ATM-mediated chromosomal break repair
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
Published in Nature neuroscience (01.09.2017)
Published in Nature neuroscience (01.09.2017)
Get full text
Journal Article
LRP-1-mediated intracellular antibody delivery to the Central Nervous System
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
Published in Scientific reports (20.07.2015)
Published in Scientific reports (20.07.2015)
Get full text
Journal Article
Encapsulation of Biomacromolecules within Polymersomes by Electroporation
Wang, Linge, Chierico, Luca, Little, Daniel, Patikarnmonthon, Nisa, Yang, Zhou, Azzouz, Mimoun, Madsen, Jeppe, Armes, Steven P., Battaglia, Giuseppe
Published in Angewandte Chemie International Edition (29.10.2012)
Published in Angewandte Chemie International Edition (29.10.2012)
Get full text
Journal Article
Adaptor protein complex 4 deficiency: a paradigm of childhood-onset hereditary spastic paraplegia caused by defective protein trafficking
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
Published in Human molecular genetics (15.01.2020)
Published in Human molecular genetics (15.01.2020)
Get full text
Journal Article
Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy
Valori, Chiara F, Ning, Ke, Wyles, Matthew, Mead, Richard J, Grierson, Andrew J, Shaw, Pamela J, Azzouz, Mimoun
Published in Science translational medicine (09.06.2010)
Published in Science translational medicine (09.06.2010)
Get more information
Journal Article
SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits
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.
Published in Nature communications (05.07.2017)
Published in Nature communications (05.07.2017)
Get full text
Journal Article
Proteinopathies as Hallmarks of Impaired Gene Expression, Proteostasis and Mitochondrial Function in Amyotrophic Lateral Sclerosis
Benson, Bridget C, Shaw, Pamela J, Azzouz, Mimoun, Highley, J Robin, Hautbergue, Guillaume M
Published in Frontiers in neuroscience (23.12.2021)
Published in Frontiers in neuroscience (23.12.2021)
Get full text
Journal Article
Towards 3D Bioprinted Spinal Cord Organoids
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
Published in International journal of molecular sciences (21.05.2022)
Published in International journal of molecular sciences (21.05.2022)
Get full text
Journal Article
Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9
Hudák, Anett, Roach, Matthew, Pusztai, Dávid, Pettkó-Szandtner, Aladár, Letoha, Annamária, Szilák, László, Azzouz, Mimoun, Letoha, Tamás
Published in International journal of molecular sciences (01.02.2023)
Published in International journal of molecular sciences (01.02.2023)
Get full text
Journal Article
Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model
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
Published in Nature medicine (01.04.2005)
Published in Nature medicine (01.04.2005)
Get full text
Journal Article
C9ORF72 hexanucleotide repeat exerts toxicity in a stable, inducible motor neuronal cell model, which is rescued by partial depletion of Pten
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
Published in Human molecular genetics (15.03.2017)
Published in Human molecular genetics (15.03.2017)
Get full text
Journal Article
Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia
Jarraya, Béchir, Boulet, Sabrina, Ralph, G Scott, Jan, Caroline, Bonvento, Gilles, Azzouz, Mimoun, Miskin, James E, Shin, Masahiro, Delzescaux, Thierry, Drouot, Xavier, Hérard, Anne-Sophie, Day, Denise M, Brouillet, Emmanuel, Kingsman, Susan M, Hantraye, Philippe, Mitrophanous, Kyriacos A, Mazarakis, Nicholas D, Palfi, Stéphane
Published in Science translational medicine (14.10.2009)
Published in Science translational medicine (14.10.2009)
Get more information
Journal Article
SRSF1-dependent inhibition of C9ORF72-repeat RNA nuclear export: genome-wide mechanisms for neuroprotection in amyotrophic lateral sclerosis
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
Published in Molecular neurodegeneration (10.08.2021)
Published in Molecular neurodegeneration (10.08.2021)
Get full text
Journal Article
Plastin 3 Promotes Motor Neuron Axonal Growth and Extends Survival in a Mouse Model of Spinal Muscular Atrophy
Alrafiah, Aziza, Karyka, Evangelia, Coldicott, Ian, Iremonger, Kayleigh, Lewis, Katherin E, Ning, Ke, Azzouz, Mimoun
Published in Molecular therapy. Methods & clinical development (15.06.2018)
Published in Molecular therapy. Methods & clinical development (15.06.2018)
Get full text
Journal Article
Optimised and rapid pre-clinical screening in the SOD1(G93A) transgenic mouse model of amyotrophic lateral sclerosis (ALS)
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
Published in PloS one (2011)
Published in PloS one (2011)
Get full text
Journal Article
Loss of TMEM106B exacerbates C9ALS/FTD DPR pathology by disrupting autophagosome maturation
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
Published in Frontiers in cellular neuroscience (16.12.2022)
Published in Frontiers in cellular neuroscience (16.12.2022)
Get full text
Journal Article
Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS
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
Published in Molecular therapy. Nucleic acids (07.09.2018)
Published in Molecular therapy. Nucleic acids (07.09.2018)
Get full text
Journal Article