Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy.

Animals Dependovirus / genetics Genetic Therapy NADH Dehydrogenase / genetics Optic Atrophy, Hereditary, Leber / genetics Tomography, Optical Coherence Visual Acuity Visual Fields

Journal

Science translational medicine
ISSN: 1946-6242
Titre abrégé: Sci Transl Med
Pays: United States
ID NLM: 101505086

Informations de publication

Date de publication:
09 12 2020
Historique:
received: 05 10 2019
revised: 17 03 2020
accepted: 02 09 2020
entrez: 10 12 2020
pubmed: 11 12 2020
medline: 24 6 2021
Statut: ppublish

Résumé

REVERSE is a randomized, double-masked, sham-controlled, multicenter, phase 3 clinical trial that evaluated the efficacy of a single intravitreal injection of rAAV2/2-

Identifiants

DOI: 10.1126/scitranslmed.aaz7423 PMID: 33298565
pubmed: 33298565
pii: 12/573/eaaz7423
doi: 10.1126/scitranslmed.aaz7423
pii:
doi:

Substances chimiques

NADH Dehydrogenase EC 1.6.99.3

Types de publication

Journal Article Multicenter Study Randomized Controlled Trial Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Medical Research Council
ID : G0701386
Pays : United Kingdom
Organisme : Medical Research Council
ID : G1002570
Pays : United Kingdom

Informations de copyright

Copyright © 2020 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.

Auteurs

Patrick 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.
Cambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge CB2 0QQ, UK.
Moorfields Eye Hospital, London EC1V 2PD, UK.
UCL Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
ORCID: 0000-0001-7847-9320

Nancy J Newman (NJ)

Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA.

Valerio Carelli (V)

IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, 40139 Bologna, Italy.
Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, 40139 Bologna, Italy.
ORCID: 0000-0003-4923-6404

Mark L Moster (ML)

Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA 19107, USA.

Valerie Biousse (V)

Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA.

Alfredo A Sadun (AA)

Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA.

Thomas Klopstock (T)

Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, 80336 Munich, Germany.
German Center for Neurodegenerative Diseases (DZNE), 80336 Munich, Germany.
Munich Cluster for Systems Neurology (SyNergy), 80336 Munich, Germany.
ORCID: 0000-0003-2805-4652

Catherine Vignal-Clermont (C)

Department of Neuro-Ophthalmology and Emergencies, Rothschild Foundation Hospital, 75019 Paris, France.
Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, FOReSIGHT, INSERM-DGOS CIC 1423, 75012 Paris, France.
ORCID: 0000-0003-0292-8905

Robert C Sergott (RC)

Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA 19107, USA.
ORCID: 0000-0001-6926-124X

Günther Rudolph (G)

Department of Ophthalmology, University Hospital, LMU Munich, 80336 Munich, Germany.
ORCID: 0000-0002-9399-730X

Chiara La Morgia (C)

IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, 40139 Bologna, Italy.
Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, 40139 Bologna, Italy.
ORCID: 0000-0002-4639-8929

Rustum Karanjia (R)

Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA.
Ottawa Hospital Research Institute and University of Ottawa Eye Institute, Ottawa, Ontario K1H 8L6, Canada.

Magali Taiel (M)

GenSight Biologics, 75012 Paris, France.

Laure Blouin (L)

GenSight Biologics, 75012 Paris, France.

Pierre Burguière (P)

GenSight Biologics, 75012 Paris, France.
ORCID: 0000-0001-9106-0786

Gerard Smits (G)

GenSight Biologics, New York, NY 10016, USA.

Caroline Chevalier (C)

GenSight Biologics, 75012 Paris, France.

Harvey Masonson (H)

GenSight Biologics, New York, NY 10016, USA.

Yordak Salermo (Y)

GenSight Biologics, New York, NY 10016, USA.

Barrett Katz (B)

GenSight Biologics, New York, NY 10016, USA.
ORCID: 0000-0003-0953-7675

Serge Picaud (S)

Sorbonne Université, INSERM, CNRS, Institut de la Vision, 75012 Paris, France.
ORCID: 0000-0002-0548-5145

David J Calkins (DJ)

The Vanderbilt Eye Institute, Vanderbilt University Medical Center, Nashville, TN 37232, USA.

José-Alain Sahel (JA)

Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, FOReSIGHT, INSERM-DGOS CIC 1423, 75012 Paris, France. sahelja@upmc.edu py237@cam.ac.uk.
Sorbonne Université, INSERM, CNRS, Institut de la Vision, 75012 Paris, France.
Fondation Ophtalmologique A. de Rothschild, 25-29 Rue Manin, 75019 Paris, France.
Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA.
ORCID: 0000-0002-4831-1153

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Bilateral visual improvement with unilateral...
questionsmedicales.fr › Virus › Virus à ADN › Parvoviridae › Parvovirinae › Dependovirus › Bilateral visual improvement with unilateral...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Bilateral visual improvement with unilateral...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Métalloprotéines › Protéines de liaison au fer › Protéines à fer non hème › Ferrosulfoprotéines › Complexe I de la chaîne respiratoire › NADH dehydrogenase › Bilateral visual improvement with unilateral...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Maladies mitochondriales › Atrophie optique héréditaire de Leber › Bilateral visual improvement with unilateral...
questionsmedicales.fr › Techniques d'investigation › Imagerie optique › Tomographie optique › Tomographie par cohérence optique › Bilateral visual improvement with unilateral...
questionsmedicales.fr › Phénomènes physiologiques oculaires › Acuité visuelle › Bilateral visual improvement with unilateral...
questionsmedicales.fr › Phénomènes physiologiques oculaires › Champs visuels › Bilateral visual improvement with unilateral...