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U7 small nuclear RNA splice-switching therapeutics for STMN2 and UNC13A in Amyotrophic Lateral Sclerosis.

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Title: U7 small nuclear RNA splice-switching therapeutics for STMN2 and UNC13A in Amyotrophic Lateral Sclerosis.
Authors: Mehta PR; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.; These authors contributed equally., Solomon T; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.; These authors contributed equally., Pickles S; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.; These authors contributed equally., Harley P; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Barioglio M; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Schweingruber C; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK., Marrero-Gagliardi A; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Gao Y; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK., Mattedi F; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Barattucci S; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Lin LT; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA., Ryadnov E; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Zanovello M; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Cammack AJ; Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.; UK Dementia Research Institute at UCL, London, UK., Isaacs AM; Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.; UK Dementia Research Institute at UCL, London, UK., Burrone J; Centre for Developmental Neurobiology and MRC Centre for Neurodevelopmental Disorders, King's College London, London, UK., Shaw CE; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK., Keuss MJ; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK., Petrucelli L; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA., Fratta P; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.; Francis Crick Institute, London, UK., Ruepp MD; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.
Source: BioRxiv : the preprint server for biology [bioRxiv] 2025 Nov 30. Date of Electronic Publication: 2025 Nov 30.
Publication Type: Journal Article; Preprint
Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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  Data: U7 small nuclear RNA splice-switching therapeutics for STMN2 and UNC13A in Amyotrophic Lateral Sclerosis.
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  Data: <searchLink fieldCode="AU" term="%22Mehta+PR%22">Mehta PR</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.; These authors contributed equally.<br /><searchLink fieldCode="AU" term="%22Solomon+T%22">Solomon T</searchLink>; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.; These authors contributed equally.<br /><searchLink fieldCode="AU" term="%22Pickles+S%22">Pickles S</searchLink>; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.; These authors contributed equally.<br /><searchLink fieldCode="AU" term="%22Harley+P%22">Harley P</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Barioglio+M%22">Barioglio M</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Schweingruber+C%22">Schweingruber C</searchLink>; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Marrero-Gagliardi+A%22">Marrero-Gagliardi A</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Gao+Y%22">Gao Y</searchLink>; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Mattedi+F%22">Mattedi F</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Barattucci+S%22">Barattucci S</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Lin+LT%22">Lin LT</searchLink>; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Ryadnov+E%22">Ryadnov E</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Zanovello+M%22">Zanovello M</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Cammack+AJ%22">Cammack AJ</searchLink>; Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.; UK Dementia Research Institute at UCL, London, UK.<br /><searchLink fieldCode="AU" term="%22Isaacs+AM%22">Isaacs AM</searchLink>; Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.; UK Dementia Research Institute at UCL, London, UK.<br /><searchLink fieldCode="AU" term="%22Burrone+J%22">Burrone J</searchLink>; Centre for Developmental Neurobiology and MRC Centre for Neurodevelopmental Disorders, King's College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Shaw+CE%22">Shaw CE</searchLink>; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Keuss+MJ%22">Keuss MJ</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Petrucelli+L%22">Petrucelli L</searchLink>; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Fratta+P%22">Fratta P</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.; Francis Crick Institute, London, UK.<br /><searchLink fieldCode="AU" term="%22Ruepp+MD%22">Ruepp MD</searchLink>; UK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.
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  Data: <searchLink fieldCode="JN" term="%22101680187%22">BioRxiv : the preprint server for biology</searchLink> [bioRxiv] 2025 Nov 30. <i>Date of Electronic Publication: </i>2025 Nov 30.
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