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

作者信息

Yee Tiffany, Wert Katherine J

机构信息

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

Peter O'Donnell Jr. Brain Institute, UT Southwestern Medical Center, Dallas, TX 75390, USA.

出版信息

Mol Ther Methods Clin Dev. 2023 Sep 3;30:573-575. doi: 10.1016/j.omtm.2023.08.019. eCollection 2023 Sep 14.

Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6670/10485576/302025d9cb11/gr1.jpg

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本文引用的文献

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CRISPR-Cas9 correction of a nonsense mutation in rescues lebercilin expression and localization in human retinal organoids.
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Leber's Congenital Amaurosis: Current Concepts of Genotype-Phenotype Correlations.
Genes (Basel). 2021 Aug 19;12(8):1261. doi: 10.3390/genes12081261.
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Organoids for the Study of Retinal Development and Developmental Abnormalities.
Front Cell Neurosci. 2021 May 5;15:667880. doi: 10.3389/fncel.2021.667880. eCollection 2021.
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Treatment Potential for LCA5-Associated Leber Congenital Amaurosis.
Invest Ophthalmol Vis Sci. 2020 May 11;61(5):30. doi: 10.1167/iovs.61.5.30.
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Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.
Mol Ther. 2018 Jun 6;26(6):1581-1593. doi: 10.1016/j.ymthe.2018.03.015. Epub 2018 Mar 21.
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Leber congenital amaurosis: genes, proteins and disease mechanisms.
Prog Retin Eye Res. 2008 Jul;27(4):391-419. doi: 10.1016/j.preteyeres.2008.05.003. Epub 2008 Jun 1.

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