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Aromatic l-amino acid decarboxylase deficiency: a patient-derived neuronal model for precision therapies.
Rossignoli, Giada; Krämer, Karolin; Lugarà, Eleonora; Alrashidi, Haya; Pope, Simon; De La Fuente Barrigon, Carmen; Barwick, Katy; Bisello, Giovanni; Ng, Joanne; Counsell, John; Lignani, Gabriele; Heales, Simon J R; Bertoldi, Mariarita; Barral, Serena; Kurian, Manju A.
Afiliación
  • Rossignoli G; Developmental Neurosciences, GOS Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Krämer K; Biological Chemistry, NBM Department, University of Verona, 37134 Verona, Italy.
  • Lugarà E; Developmental Neurosciences, GOS Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Alrashidi H; Clinical and Experimental Epilepsy, Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.
  • Pope S; Genetics and Genomic Medicine, GOS Institute of Child Health, University College London, London WC1N 1EH, UK.
  • De La Fuente Barrigon C; Neurometabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London WC1N 3BG, UK.
  • Barwick K; Genetics and Genomic Medicine, GOS Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Bisello G; Developmental Neurosciences, GOS Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Ng J; Biological Chemistry, NBM Department, University of Verona, 37134 Verona, Italy.
  • Counsell J; Developmental Neurosciences, GOS Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Lignani G; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London WC1E 6HU, UK.
  • Heales SJR; Developmental Neurosciences, GOS Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Bertoldi M; Clinical and Experimental Epilepsy, Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.
  • Barral S; Neurometabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London WC1N 3BG, UK.
  • Kurian MA; Centre for Inborn Errors of Metabolism, GOS Institute of Child Health, UniversCity College London, London WC1N 1EH, UK.
Brain ; 144(8): 2443-2456, 2021 09 04.
Article en En | MEDLINE | ID: mdl-33734312
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Descarboxilasas de Aminoácido-L-Aromático / Dopaminérgicos / Levodopa / Neurogénesis / Células Madre Pluripotentes Inducidas / Errores Innatos del Metabolismo de los Aminoácidos / Neuronas Tipo de estudio: Prognostic_studies Idioma: En Revista: Brain Año: 2021 Tipo del documento: Article
Texto completo
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Descarboxilasas de Aminoácido-L-Aromático / Dopaminérgicos / Levodopa / Neurogénesis / Células Madre Pluripotentes Inducidas / Errores Innatos del Metabolismo de los Aminoácidos / Neuronas Tipo de estudio: Prognostic_studies Idioma: En Revista: Brain Año: 2021 Tipo del documento: Article