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Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
Le Meur, Guylène; Lebranchu, Pierre; Billaud, Fanny; Adjali, Oumeya; Schmitt, Sébastien; Bézieau, Stéphane; Péréon, Yann; Valabregue, Romain; Ivan, Catherine; Darmon, Christophe; Moullier, Philippe; Rolling, Fabienne; Weber, Michel.
Afiliación
  • Le Meur G; Ophthalmology Department, University Hospital Centre (CHU) de Nantes, Nantes, France; INSERM UMR 1089, University of Nantes, CHU de Nantes, Nantes France. Electronic address: guylene.lemeur@univ-nantes.fr.
  • Lebranchu P; Ophthalmology Department, University Hospital Centre (CHU) de Nantes, Nantes, France; UMR 6597 CNRS, Image and Video Communication Team, Institute for Research into Communications and Cybernetics of Nantes, Polytech Nantes, Nantes, France.
  • Billaud F; Ophthalmology Department, University Hospital Centre (CHU) de Nantes, Nantes, France.
  • Adjali O; INSERM UMR 1089, University of Nantes, CHU de Nantes, Nantes France.
  • Schmitt S; Department of Genetics, CHU de Nantes, Nantes, France.
  • Bézieau S; Department of Genetics, CHU de Nantes, Nantes, France.
  • Péréon Y; Reference Centre for Neuromuscular Disorders, FILNEMUS, CHU de Nantes, Nantes, France.
  • Valabregue R; Institut du Cerveau et de la Moelle épinière ICM, Centre for NeuroImaging Research (CENIR), Paris, France.
  • Ivan C; Ophthalmology Department, University Hospital Centre (CHU) de Nantes, Nantes, France.
  • Darmon C; INSERM UMR 1089, University of Nantes, CHU de Nantes, Nantes France.
  • Moullier P; INSERM UMR 1089, University of Nantes, CHU de Nantes, Nantes France.
  • Rolling F; INSERM UMR 1089, University of Nantes, CHU de Nantes, Nantes France.
  • Weber M; Ophthalmology Department, University Hospital Centre (CHU) de Nantes, Nantes, France; INSERM UMR 1089, University of Nantes, CHU de Nantes, Nantes France.
Mol Ther ; 26(1): 256-268, 2018 01 03.
Article en En | MEDLINE | ID: mdl-29033008

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Dependovirus / Cis-trans-Isomerasas / Amaurosis Congénita de Leber / Vectores Genéticos Tipo de estudio: Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Child / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Dependovirus / Cis-trans-Isomerasas / Amaurosis Congénita de Leber / Vectores Genéticos Tipo de estudio: Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Child / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos