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http://dx.doi.org/10.1016/j.omtm.2023.08.019 | DOI Listing |
Mol Ther Methods Clin Dev
September 2023
Department of Ophthalmology, UT Southwestern Medical Center, Dallas, TX 75390, USA.
Methods Mol Biol
March 2022
UCL Institute of Ophthalmology, London, UK.
Front Neurosci
April 2017
Department of Ophthalmology, Leiden University Medical CenterLeiden, Netherlands.
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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