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

Autor: Günther Rudolph, Magali Taiel, Laure Blouin, Nancy J. Newman, Rustum Karanjia, Catherine Vignal-Clermont, Thomas Klopstock, Serge Picaud, Robert C. Sergott, Valérie Biousse, Gerard Smits, Caroline Chevalier, Mark L. Moster, Valerio Carelli, Chiara La Morgia, Alfredo A. Sadun, Harvey Masonson, Patrick Yu-Wai-Man, Barrett Katz, David J. Calkins, Yordak Salermo, Pierre Burguière, José-Alain Sahel

Publikováno v: Science Translational Medicine
Science Translational Medicine, American Association for the Advancement of Science, 2020, 12 (573), ⟨10.1126/scitranslmed.aaz7423⟩
Science Translational Medicine, 2020, 12 (573), ⟨10.1126/scitranslmed.aaz7423⟩
Science translational medicine 12(573), eaaz7423 (2020). doi:10.1126/scitranslmed.aaz7423

International audience; 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-ND4 in subjects with visual loss from Leber hereditary opti

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1e8b2819dfe0c1739c28bb816ac645e4
https://hal.sorbonne-universite.fr/hal-03373492/document