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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
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  Data: Large-scale drug screening in iPSC-derived motor neurons from sporadic ALS patients identifies a potential combinatorial therapy.
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  Data: <searchLink fieldCode="AU" term="%22Bye+CR%22">Bye CR</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia. chris.bye@florey.edu.au.<br /><searchLink fieldCode="AU" term="%22Qian+E%22">Qian E</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Lim+K%22">Lim K</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Daniszewski+M%22">Daniszewski M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Garton+FC%22">Garton FC</searchLink>; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Trần-Lê+BC%22">Trần-Lê BC</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Liang+HH%22">Liang HH</searchLink>; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Lin+T%22">Lin T</searchLink>; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Lock+JG%22">Lock JG</searchLink>; School of Biomedical Sciences, University of New South Wales, Sydney, New South Wales, Australia.<br /><searchLink fieldCode="AU" term="%22Crombie+DE%22">Crombie DE</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Morgan+S%22">Morgan S</searchLink>; Melbourne Bioinformatics, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Hu+Y%22">Hu Y</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Barton+SK%22">Barton SK</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Palmer+LM%22">Palmer LM</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Djouma+E%22">Djouma E</searchLink>; Department of Microbiology, Anatomy, Physiology and Pharmacology, La Trobe University, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Kodikara+S%22">Kodikara S</searchLink>; Melbourne Integrative Genomics, School of Mathematics and Statistics, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Lê+Cao+KA%22">Lê Cao KA</searchLink>; Melbourne Integrative Genomics, School of Mathematics and Statistics, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Dharmadasa+T%22">Dharmadasa T</searchLink>; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Henders+AK%22">Henders AK</searchLink>; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Ziser+LA%22">Ziser LA</searchLink>; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Kiernan+MC%22">Kiernan MC</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Talbot+K%22">Talbot K</searchLink>; Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.<br /><searchLink fieldCode="AU" term="%22Needham+M%22">Needham M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Fletcher+S%22">Fletcher S</searchLink>; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth, Western Australia, Australia.<br /><searchLink fieldCode="AU" term="%22Talman+P%22">Talman P</searchLink>; Neurosciences Department, University Hospital Geelong, Geelong, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Mathers+S%22">Mathers S</searchLink>; Calvary Healthcare Bethlehem Hospital Neuro-Consultancy, South Caulfield, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Wray+NR%22">Wray NR</searchLink>; Institute for Molecular Bioscience, The University of Queensland, Brisbane, Queensland, Australia.; Queensland Brain Institute, The University of Queensland, Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Hewitt+AW%22">Hewitt AW</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Pebay+A%22">Pebay A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Turner+BJ%22">Turner BJ</searchLink>; 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.
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  Data: <searchLink fieldCode="JN" term="%229809671%22">Nature neuroscience</searchLink> [Nat Neurosci] 2026 Jan; Vol. 29 (1), pp. 40-52. <i>Date of Electronic Publication: </i>2025 Nov 24.
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