Recovery of Cone-Mediated Vision in Lebercilin-Associated Severe Retinal Ciliopathy (LCA5) after Gene Therapy: One Year Results of a Phase Ib/IIa Trial.
Aleman TS, Uyhazi KE, Roman AJ, Weber ML, O'Neil EC, Swider M, Sumaroka A, Maguire KH, Aleman EM, Santos AJ, Kim RJ, Parchinski KM, Billek A, Fradin M, Chung W, Margaritis P, Sun J, Scoles DH, Wu V, Garafalo AV, Jayagopal A, Yerxa B, Tuller S, Maguire AM, Bennett J, Cideciyan AV.
Aleman TS, et al. Among authors: roman aj.
Mol Ther. 2025 Jul 1:S1525-0016(25)00486-1. doi: 10.1016/j.ymthe.2025.06.035. Online ahead of print.
Mol Ther. 2025.
PMID: 40598770