Creation of de novo cryptic splicing for ALS and FTD precision medicine.

Autor: Wilkins OG; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.; The Francis Crick Institute, London NW1 1AT, UK., Chien MZYJ; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.; The Francis Crick Institute, London NW1 1AT, UK., Wlaschin JJ; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.; Department of Biology, Johns Hopkins University, Baltimore, MD 21218, USA., Barattucci S; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Harley P; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Mattedi F; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Mehta PR; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Pisliakova M; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.; The Francis Crick Institute, London NW1 1AT, UK., Ryadnov E; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Keuss MJ; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Thompson D; Mammalian Genetics Unit, MRC Harwell Institute, Oxfordshire OX11 0RD, UK., Digby H; The Francis Crick Institute, London NW1 1AT, UK.; UK Dementia Research Institute at King's College London, London SE5 9RX, UK., Knez L; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.; The Francis Crick Institute, London NW1 1AT, UK., Simkin RL; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Diaz JA; EGA-Institute for Women's Health, University College London, London WC1E 6HX, UK., Zanovello M; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.; The Francis Crick Institute, London NW1 1AT, UK., Brown AL; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Darbey A; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Karda R; EGA-Institute for Women's Health, University College London, London WC1E 6HX, UK., Fisher EMC; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Cunningham TJ; Mammalian Genetics Unit, MRC Harwell Institute, Oxfordshire OX11 0RD, UK.; MRC Prion Unit at UCL and UCL Institute of Prion Diseases, London W1W 7FF, UK., Le Pichon CE; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA., Ule J; The Francis Crick Institute, London NW1 1AT, UK.; UK Dementia Research Institute at King's College London, London SE5 9RX, UK., Fratta P; UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.; The Francis Crick Institute, London NW1 1AT, UK.

Publikováno v: Science (New York, N.Y.) [Science] 2024 Oct 04; Vol. 386 (6717), pp. 61-69. Date of Electronic Publication: 2024 Oct 03.