Leber Congenital Amaurosis Due to <i>GUCY2D</i> Mutations: Longitudinal Analysis of Retinal Structure and Visual Function

Leber Congenital Amaurosis Due to <i>GUCY2D</i> Mutations: Longitudinal Analysis of Retinal Structure and Visual Function

Gene augmentation therapy is being planned for <i>GUCY2D</i>-associated Leber congenital amaurosis (LCA). To increase our understanding of the natural history of <i>GUCY2D</i>-LCA, patients were evaluated twice with an interval of 4 to 7 years between visits using safety and...

Full description

Bibliographic Details
Main Authors: Samuel G. Jacobson, Artur V. Cideciyan, Alexander Sumaroka, Alejandro J. Roman, Vivian Wu, Malgorzata Swider, Rebecca Sheplock, Arun K. Krishnan, Alexandra V. Garafalo
Format: Article
Language:English
Published: MDPI AG 2021-02-01
Series:International Journal of Molecular Sciences
Subjects:
cone
clinical trials
rod
optical coherence tomography
outcome measures
Online Access:https://www.mdpi.com/1422-0067/22/4/2031

Similar Items

Similar Items