The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy

Inherited retinal dystrophies (IRDs) are caused by mutations in over 200 genes, resulting in a range of therapeutic options. Translational read-through inducing drugs (TRIDs) offer the possibility of treating multiple IRDs regardless of the causative gene. TRIDs promote ribosomal misreading of prema...

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Udgivet i:Sci Rep
Main Authors: Torriano, Simona, Erkilic, Nejla, Baux, David, Cereso, Nicolas, De Luca, Valerie, Meunier, Isabelle, Moosajee, Mariya, Roux, Anne-Francoise, Hamel, Christian P., Kalatzis, Vasiliki
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2018
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5974348/
https://ncbi.nlm.nih.gov/pubmed/29844446
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-018-26481-7
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