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
Description
ISSN:2692-8205
DOI:10.1101/2025.11.26.690143