Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy.
Yu-Wai-Man P, Newman NJ, Carelli V, Moster ML, Biousse V, Sadun AA, Klopstock T, Vignal-Clermont C, Sergott RC, Rudolph G, La Morgia C, Karanjia R, Taiel M, Blouin L, Burguière P, Smits G, Chevalier C, Masonson H, Salermo Y, Katz B, Picaud S, Calkins DJ, Sahel JA.
Yu-Wai-Man P, et al.
Sci Transl Med. 2020 Dec 9;12(573):eaaz7423. doi: 10.1126/scitranslmed.aaz7423.
Sci Transl Med. 2020.
PMID: 33298565
Clinical Trial.