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dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice.
Colasante, Gaia; Lignani, Gabriele; Brusco, Simone; Di Berardino, Claudia; Carpenter, Jenna; Giannelli, Serena; Valassina, Nicholas; Bido, Simone; Ricci, Raffaele; Castoldi, Valerio; Marenna, Silvia; Church, Timothy; Massimino, Luca; Morabito, Giuseppe; Benfenati, Fabio; Schorge, Stephanie; Leocani, Letizia; Kullmann, Dimitri M; Broccoli, Vania.
Afiliación
  • Colasante G; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy. Electronic address: colasante.gaia@hsr.it.
  • Lignani G; Department of Clinical and Experimental Epilepsy, UCL Institute of Neurology, University College London, Queen Square, London WC1N 3BG, UK.
  • Brusco S; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Di Berardino C; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Carpenter J; Department of Clinical and Experimental Epilepsy, UCL Institute of Neurology, University College London, Queen Square, London WC1N 3BG, UK.
  • Giannelli S; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Valassina N; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Bido S; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Ricci R; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Castoldi V; Experimental Neurophysiology Unit, Institute of Experimental Neurology (INSPE), San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Marenna S; Experimental Neurophysiology Unit, Institute of Experimental Neurology (INSPE), San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Church T; Department of Clinical and Experimental Epilepsy, UCL Institute of Neurology, University College London, Queen Square, London WC1N 3BG, UK.
  • Massimino L; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Morabito G; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Benfenati F; Center for Synaptic Neuroscience and Technology, Istituto Italiano di Tecnologia, 16132 Genova, Italy; IRCCS Ospedale Policlinico San Martino, University of Genova, 16132 Genova, Italy.
  • Schorge S; Department of Clinical and Experimental Epilepsy, UCL Institute of Neurology, University College London, Queen Square, London WC1N 3BG, UK.
  • Leocani L; Experimental Neurophysiology Unit, Institute of Experimental Neurology (INSPE), San Raffaele Scientific Institute, 20132 Milan, Italy.
  • Kullmann DM; Department of Clinical and Experimental Epilepsy, UCL Institute of Neurology, University College London, Queen Square, London WC1N 3BG, UK.
  • Broccoli V; Stem Cell and Neurogenesis Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy; CNR Institute of Neuroscience, 20129 Milan, Italy. Electronic address: broccoli.vania@hsr.it.
Mol Ther ; 28(1): 235-253, 2020 01 08.
Article en En | MEDLINE | ID: mdl-31607539
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Convulsiones / Terapia Genética / Activación Transcripcional / Epilepsias Mioclónicas / Canal de Sodio Activado por Voltaje NAV1.1 / Proteína 9 Asociada a CRISPR / Interneuronas Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Convulsiones / Terapia Genética / Activación Transcripcional / Epilepsias Mioclónicas / Canal de Sodio Activado por Voltaje NAV1.1 / Proteína 9 Asociada a CRISPR / Interneuronas Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos