Human retina-in-a-dish: Unlocking the potential to study mechanisms of inherited retinal disease.

Mol Ther Methods Clin Dev

Department of Ophthalmology, UT Southwestern Medical Center, Dallas, TX 75390, USA.

Published: September 2023

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10485576PMC
http://dx.doi.org/10.1016/j.omtm.2023.08.019DOI Listing

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Article Synopsis
  • Inherited retinal dystrophies like Leber congenital amaurosis and retinitis pigmentosa involve the loss of photoreceptors and have limited treatment options.
  • Advances in induced pluripotent stem cell technology allow researchers to create retinal organoids, which serve as models for studying retinal development and disease mechanisms.
  • These 3D cultures can be used to test potential therapies, such as antisense oligonucleotides, by mimicking patient mutations and assessing treatment effectiveness in a controlled lab setting.
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Once considered science fiction, gene therapy is rapidly becoming scientific reality, targeting a growing number of the approximately 250 genes linked to hereditary retinal disorders such as retinitis pigmentosa and Leber's congenital amaurosis. Powerful new technologies have emerged, leading to the development of humanized models for testing and screening these therapies, bringing us closer to the goal of personalized medicine. These tools include the ability to differentiate human induced pluripotent stem cells (iPSCs) to create a "retina-in-a-dish" model and the self-formed ectodermal autonomous multi-zone, which can mimic whole eye development.

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