Gene-agnostic approaches to treating inherited retinal degenerations.

ABSTRACT: Most patients with inherited retinal degenerations (IRDs) have been waiting for treatments that are "just around the corner" for decades, with only a handful of seminal breakthroughs happening in recent years. Highlighting the difficulties in the quest for curative therapeutics, Luxturna required 16 years of development before finally obtaining United States Food and Drug Administration (FDA) approval and its international equivalents. IRDs are both genetically and phenotypically heterogeneous. While this diversity offers many opportunities for gene-by-gene precision medicine-based approaches, it also poses a significant challenge. For this reason, alternative (or parallel) strategies to identify more comprehensive, across-the-board therapeutics for the genetically and phenotypically diver

SUBMITTER: Chew LA 

PROVIDER: S-EPMC10133473 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature