Large-scale drug screening in iPSC-derived motor neurons from sporadic ALS patients identifies a potential combinatorial therapy.

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Title: Large-scale drug screening in iPSC-derived motor neurons from sporadic ALS patients identifies a potential combinatorial therapy.
Authors: Bye CR; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia. chris.bye@florey.edu.au., Qian E; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Lim K; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Daniszewski M; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, Victoria, Australia.; Department of Anatomy and Physiology, The University of Melbourne, Melbourne, Victoria, Australia., Garton FC; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia., Trần-Lê BC; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Liang HH; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, Victoria, Australia., Lin T; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia., Lock JG; School of Biomedical Sciences, University of New South Wales, Sydney, New South Wales, Australia., Crombie DE; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Morgan S; Melbourne Bioinformatics, The University of Melbourne, Melbourne, Victoria, Australia., Hu Y; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Barton SK; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Palmer LM; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Djouma E; Department of Microbiology, Anatomy, Physiology and Pharmacology, La Trobe University, Melbourne, Victoria, Australia., Kodikara S; Melbourne Integrative Genomics, School of Mathematics and Statistics, The University of Melbourne, Melbourne, Victoria, Australia., Lê Cao KA; Melbourne Integrative Genomics, School of Mathematics and Statistics, The University of Melbourne, Melbourne, Victoria, Australia., Dharmadasa T; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia., Henders AK; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia., Ziser LA; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia., Kiernan MC; Neuroscience Research Australia, University of New South Wales, Sydney, New South Wales, Australia.; Neurology Department, South Eastern Sydney Local Health District, Sydney, New South Wales, Australia., Talbot K; Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK., Needham M; Department of Neurology, Fiona Stanley Hospital, Perth, Western Australia, Australia.; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth, Western Australia, Australia.; Notre Dame University, Fremantle, Western Australia, Australia., Fletcher S; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth, Western Australia, Australia., Talman P; Neurosciences Department, University Hospital Geelong, Geelong, Victoria, Australia., Mathers S; Calvary Healthcare Bethlehem Hospital Neuro-Consultancy, South Caulfield, Victoria, Australia., Wray NR; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia.; Queensland Brain Institute, The University of Queensland, Brisbane, Queensland, Australia., Hewitt AW; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, Victoria, Australia.; Menzies Institute for Medical Research, University of Tasmania, Hobart, Tasmania, Australia., Pebay A; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, Victoria, Australia.; Department of Anatomy and Physiology, The University of Melbourne, Melbourne, Victoria, Australia.; Department of Surgery, Royal Melbourne Hospital, The University of Melbourne, Melbourne, Victoria, Australia., Turner BJ; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia. bradley.turner@florey.edu.au.; Perron Institute for Neurological and Translational Science, Perth, Western Australia, Australia. bradley.turner@florey.edu.au.
Source: Nature neuroscience [Nat Neurosci] 2026 Jan; Vol. 29 (1), pp. 40-52. Date of Electronic Publication: 2025 Nov 24.
Publication Type: Journal Article
Journal Info: Publisher: Nature Publishing Group Country of Publication: United States NLM ID: 9809671 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-1726 (Electronic) Linking ISSN: 10976256 NLM ISO Abbreviation: Nat Neurosci Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1546-1726
DOI:10.1038/s41593-025-02118-7