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Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial.
Tardieu, Marc; Zérah, Michel; Gougeon, Marie-Lise; Ausseil, Jérome; de Bournonville, Stéphanie; Husson, Béatrice; Zafeiriou, Dimitrios; Parenti, Giancarlo; Bourget, Philippe; Poirier, Béatrice; Furlan, Valérie; Artaud, Cécile; Baugnon, Thomas; Roujeau, Thomas; Crystal, Ronald G; Meyer, Christian; Deiva, Kumaran; Heard, Jean-Michel.
Afiliación
  • Tardieu M; Paediatric Neurology Department, Université Paris Sud and Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Sud, Le Kremlin-Bicêtre, France. Electronic address: marc.tardieu@aphp.fr.
  • Zérah M; Paediatric Neurosurgery Department, Université Paris Descartes and Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker, Paris, France.
  • Gougeon ML; Antiviral Immunity, Biotherapy and Vaccine unit, Infection and Epidemiology Department, Institut Pasteur, Paris, France.
  • Ausseil J; Laboratoire de Biochimie and INSERM U1088, Université de Picardie-Jules Verne, Hôpitaux Universitaires d'Amiens, Amiens, France.
  • de Bournonville S; Paediatric Neurology Department, Université Paris Sud and Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Sud, Le Kremlin-Bicêtre, France.
  • Husson B; Paediatric Radiology Department, Université Paris Sud and Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Sud, Le Kremlin-Bicêtre, France.
  • Zafeiriou D; Department of Paediatrics, Aristotle University, Thessaloniki, Greece.
  • Parenti G; Department of Translational Medical Sciences, Frederico II University, Naples, Italy; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Bourget P; Clinical Pharmacy Department, Université Paris Descartes and Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker, Paris, France.
  • Poirier B; Antiviral Immunity, Biotherapy and Vaccine unit, Infection and Epidemiology Department, Institut Pasteur, Paris, France.
  • Furlan V; Pharmacology Toxicology Department, Université Paris Sud and Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Sud, Le Kremlin-Bicêtre, France.
  • Artaud C; Centre for Translational Science, Clinical Core, Institut Pasteur, Paris, France.
  • Baugnon T; Anaesthesiology Department, Université Paris Descartes and Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker, Paris, France.
  • Roujeau T; Neuroscience Department, Hôpitaux de Montpellier, Montpellier, France.
  • Crystal RG; Department of Genetic Medicine, Weill Cornell Medical College, New York, NY, USA.
  • Meyer C; UniQure, Amsterdam, Netherlands.
  • Deiva K; Paediatric Neurology Department, Université Paris Sud and Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Sud, Le Kremlin-Bicêtre, France.
  • Heard JM; Department of Neuroscience, Biotherapy and Neurodegenerative Diseases Unit, INSERM U1115, Institut Pasteur, Paris, France.
Lancet Neurol ; 16(9): 712-720, 2017 09.
Article en En | MEDLINE | ID: mdl-28713035
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Acetilglucosaminidasa / Encéfalo / Terapia Genética / Evaluación de Resultado en la Atención de Salud / Mucopolisacaridosis III / Dependovirus / Vectores Genéticos Tipo de estudio: Clinical_trials Límite: Child, preschool / Humans / Infant Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2017 Tipo del documento: Article Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Acetilglucosaminidasa / Encéfalo / Terapia Genética / Evaluación de Resultado en la Atención de Salud / Mucopolisacaridosis III / Dependovirus / Vectores Genéticos Tipo de estudio: Clinical_trials Límite: Child, preschool / Humans / Infant Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2017 Tipo del documento: Article Pais de publicación: Reino Unido