Document Type
Article
Publication Date
6-20-2023
Abstract
PURPOSE: An intravitreally injected antisense oligonucleotide, sepofarsen, was designed to modulate splicing within retinas of patients with severe vision loss due to deep intronic c.2991 + 1655A > G variant in the
OBSERVATIONS: Visual function was evaluated with best corrected standard and low-luminance visual acuities, microperimetry, dark-adapted chromatic perimetry, and full-field sensitivity testing. Retinal structure was evaluated with OCT imaging. At the fovea, all visual function measures and IS/OS intensity of the OCT showed transient improvements peaking at 3-6 months, remaining better than baseline at ∼2 years, and returning to baseline by 3-4 years after each single injection.
CONCLUSIONS AND IMPORTANCE: These results suggest that sepofarsen reinjection intervals may need to be longer than 2 years.
Recommended Citation
Cideciyan, Artur V; Jacobson, Samuel G; Ho, Allen; Swider, Malgorzata; Sumaroka, Alexander; Roman, Alejandro J; Wu, Vivian; Russell, Robert C; Viarbitskaya, Iryna; Garafalo, Alexandra V; Schwartz, Michael R; and Girach, Aniz, "Durable Vision Improvement After a Single Intravitreal Treatment With Antisense Oligonucleotide in CEP290-Lca: Replication in Two Eyes" (2023). Wills Eye Hospital Papers. Paper 192.
https://jdc.jefferson.edu/willsfp/192
Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.
PubMed ID
37388818
Language
English
Comments
This article is the author's final published version in American Journal of Ophthalmology Case Reports, Volume 32, 2023, Article number 101873.
The published version is available at https://doi.org/10.1016/j.ajoc.2023.101873.
Copyright © 2023 The Authors. Published by Elsevier Inc.
This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by- nc-nd/4.0/).