Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy
Patrick Yu‐Wai‐Man, Nancy J. Newman, Valério Carelli, Mark L. Moster, Valérie Biousse, Alfredo A. Sadun, Thomas Klopstock, Catherine Vignal, Robert C. Sergott, Günther Rudolph, Chiara La Morgia, Rustum Karanjia, Magali Taiel, Laure Blouin, Pierre Burguière, Gerard Smits, Caroline Chevalier, Harvey Masonson, Yordak Salermo, Barrett Katz, Serge Picaud, David J. Calkins, José‐Alain Sahel
Abstract
= 0.894). At week 96, 25 subjects (68%) had a clinically relevant recovery in BCVA from baseline in at least one eye, and 29 subjects (78%) had an improvement in vision in both eyes. A nonhuman primate study was conducted to investigate this bilateral improvement. Evidence of transfer of viral vector DNA from the injected eye to the anterior segment, retina, and optic nerve of the contralateral noninjected eye supports a plausible mechanistic explanation for the unexpected bilateral improvement in visual function after unilateral injection.