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Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up.
Roubertie, Agathe; Opladen, Thomas; Brennenstuhl, Heiko; Kuseyri Hübschmann, Oya; Flint, Lisa; Willemsen, Michel A; Leuzzi, Vincenzo; Cazorla, Angels Garcia; Kurian, Manju A; François-Heude, Marie Céline; Hwu, Paul; Zeev, Bruria Ben; Kiening, Karl; Roujeau, Thomas; Pons, Roser; Pearson, Toni S.
Afiliação
  • Roubertie A; CHU Montpellier, Département de Neuropédiatrie, INM, Univ Montpellier, INSERM U 1298, Montpellier, France.
  • Opladen T; Division of Child Neurology and Metabolic Medicine, University Children's Hospital Heidelberg, Germany.
  • Brennenstuhl H; Division of Child Neurology and Metabolic Medicine, University Children's Hospital Heidelberg, Germany.
  • Kuseyri Hübschmann O; Institute Human Genetics, University Children's Hospital Heidelberg, Germany.
  • Flint L; Division of Child Neurology and Metabolic Medicine, University Children's Hospital Heidelberg, Germany.
  • Willemsen MA; AADC Research Trust, Caterham, UK.
  • Leuzzi V; Department of Pediatric Neurology, Donders Institute for Brain, Cognition, and Behavior, Radboud University Medical Center, Nijmegen, The Netherlands.
  • Cazorla AG; Department of Human Neuroscience-Unit of Child Neurology and Psychiatry, University of Rome La Sapienza.
  • Kurian MA; Neurometabolism Unit, Department of Neurology, CIBERER and MetabERN, Hospital Sant Joan de Déu, Barcelona, Spain.
  • François-Heude MC; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, Great Ormond Street Institute of Child Health, University College London, London, UK.
  • Hwu P; Department of Neurology, Great Ormond Street Hospital for Children, London, UK.
  • Zeev BB; CHU Montpellier, Département de Neuropédiatrie, INM, Univ Montpellier, INSERM U 1298, Montpellier, France.
  • Kiening K; Department of Pediatrics and Medical Genetics, National Taiwan University Hospital, Taipei, Taiwan.
  • Roujeau T; Pediatric Neurology Unit, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Ramat Gan, Israel.
  • Pons R; Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
  • Pearson TS; Division of Stereotactic Neurosurgery, University Hospital Heidelberg, Germany.
J Inherit Metab Dis ; 2023 Jul 04.
Article em En | MEDLINE | ID: mdl-37402126
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Guideline / Qualitative_research Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Guideline / Qualitative_research Idioma: En Ano de publicação: 2023 Tipo de documento: Article