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

• DOI: 10.1126/scitranslmed.aaz7423
• Publication Date: 2020-12-09T20:21:11Z
• Authors: Patrick Yu-Wai-Man; Nancy J. Newman; Valerio Carelli; Mark L. Moster; Valerie 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
• Keywords: 0301 basic medicine; Visual Acuity; NADH Dehydrogenase; Genetic Therapy; Optic Atrophy, Hereditary, Leber; Dependovirus; therapy [Optic Atrophy, Hereditary, Leber]; genetics [Dependovirus]; 3. Good health; gene therapy; LHON; Leber's hereditary optic neuropathy; visual improvement; 03 medical and health sciences; genetics [Optic Atrophy, Hereditary, Leber]; Animals; Humans; ddc:500; Visual Fields; genetics [NADH Dehydrogenase]; [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology; Tomography, Optical Coherence

At 96 weeks after unilateral intravitreal injection of rAAV2/2- ND4 , vision improved in both eyes 78% subjects affected with LHON.