Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes.

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 CEP290 gene. A previous report showed vision improvements following a single injection in one eye with unexpected durability lasting at least 15 months. The current study evaluated durability of efficacy beyond 15 months in the previously treated left eye. In addition, peak efficacy and durability were evaluated in the treatment-naive right eye, and re-injection of the left eye 4 years after the first injection.

Observations

Visual function was evaluated with best corrected standard and low-luminance visual acuities, microperimetry, dark-adapted chromatic pe

SUBMITTER: Cideciyan AV 

PROVIDER: S-EPMC10302566 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature