Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids

RP2 mutations cause a severe form of X-linked retinitis pigmentosa (XLRP). The mechanism of RP2-associated retinal degeneration in humans is unclear, and animal models of RP2 XLRP do not recapitulate this severe phenotype. Here, we developed gene-edited isogenic RP2 knockout (RP2 KO) induced pluripo...

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Udgivet i:Stem Cell Reports
Main Authors: Lane, Amelia, Jovanovic, Katarina, Shortall, Ciara, Ottaviani, Daniele, Panes, Anna Brugulat, Schwarz, Nele, Guarascio, Rosellina, Hayes, Matthew J., Palfi, Arpad, Chadderton, Naomi, Farrar, G. Jane, Hardcastle, Alison J., Cheetham, Michael E.
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7363745/
https://ncbi.nlm.nih.gov/pubmed/32531192
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2020.05.007
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