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Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy.
Yu-Wai-Man, Patrick; Newman, Nancy J; Carelli, Valerio; Moster, Mark L; Biousse, Valerie; Sadun, Alfredo A; Klopstock, Thomas; Vignal-Clermont, Catherine; Sergott, Robert C; Rudolph, Günther; La Morgia, Chiara; Karanjia, Rustum; Taiel, Magali; Blouin, Laure; Burguière, Pierre; Smits, Gerard; Chevalier, Caroline; Masonson, Harvey; Salermo, Yordak; Katz, Barrett; Picaud, Serge; Calkins, David J; Sahel, José-Alain.
Afiliación
  • Yu-Wai-Man P; Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0PY, UK. sahelja@upmc.edu py237@cam.ac.uk.
  • Newman NJ; Cambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge CB2 0QQ, UK.
  • Carelli V; Moorfields Eye Hospital, London EC1V 2PD, UK.
  • Moster ML; UCL Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
  • Biousse V; Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA.
  • Sadun AA; IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, 40139 Bologna, Italy.
  • Klopstock T; Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, 40139 Bologna, Italy.
  • Vignal-Clermont C; Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA 19107, USA.
  • Sergott RC; Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA.
  • Rudolph G; Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA.
  • La Morgia C; Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, 80336 Munich, Germany.
  • Karanjia R; German Center for Neurodegenerative Diseases (DZNE), 80336 Munich, Germany.
  • Taiel M; Munich Cluster for Systems Neurology (SyNergy), 80336 Munich, Germany.
  • Blouin L; Department of Neuro-Ophthalmology and Emergencies, Rothschild Foundation Hospital, 75019 Paris, France.
  • Burguière P; Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, FOReSIGHT, INSERM-DGOS CIC 1423, 75012 Paris, France.
  • Smits G; Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA 19107, USA.
  • Chevalier C; Department of Ophthalmology, University Hospital, LMU Munich, 80336 Munich, Germany.
  • Masonson H; IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, 40139 Bologna, Italy.
  • Salermo Y; Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, 40139 Bologna, Italy.
  • Katz B; Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA.
  • Picaud S; Ottawa Hospital Research Institute and University of Ottawa Eye Institute, Ottawa, Ontario K1H 8L6, Canada.
  • Calkins DJ; GenSight Biologics, 75012 Paris, France.
  • Sahel JA; GenSight Biologics, 75012 Paris, France.
Sci Transl Med ; 12(573)2020 12 09.
Article en En | MEDLINE | ID: mdl-33298565
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Atrofia Óptica Hereditaria de Leber Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Atrofia Óptica Hereditaria de Leber Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos