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New Drug Discovery Paradigms for Retinal Diseases: A Focus on Retinal Organoids.

Authors
  • Aasen, Davis M1
  • Vergara, M Natalia1, 2, 3
  • 1 Department of Ophthalmology, Sue Anschutz-Rodgers Eye Center, University of Colorado School of Medicine, Aurora, Colorado.
  • 2 CellSight Ocular Stem Cell and Regeneration Program, University of Colorado School of Medicine, Aurora, Colorado.
  • 3 Linda Crnic Institute for Down Syndrome, University of Colorado School of Medicine, Aurora, Colorado.
Type
Published Article
Journal
Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and Therapeutics
Publication Date
Jan 01, 2020
Volume
36
Issue
1
Pages
18–24
Identifiers
DOI: 10.1089/jop.2018.0140
PMID: 31059378
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

Retinal disease represents a growing global problem, both in terms of quality of life and economic impact, yet new therapies are not being developed at a sufficient rate to meet this mounting need. In this context, retinal organoids derived from human induced pluripotent stem cells hold significant promise for improving upon the current drug development process, increasing the speed and efficiency of moving potential therapeutic agents from bench to bedside. These organoid systems display the cell-cell and cell-matrix interactions, cellular heterogeneity, and physiological responses reflective of human biology and, thus, have the ability to replicate retinal disease pathology in a way that 2-dimensional cell cultures and animal models have been heretofore unable to achieve. However, organoid technology is not yet mature enough to meet the high-throughput demands of the first stages of drug screening. Hence, the augmentation of the existing drug development pipeline with retinal organoids, rather than the replacement of existing pathway components, may provide a way to harness the benefits of this improved pathological modeling. In this study, we outline the possible benefits of such a symbiosis, discuss other potential uses, and highlight barriers that remain to be overcome.

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