Article
The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy
22 May 2018
Abstract excerpt
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 premature stop codons, which results in the incorporation of a near-cognate amino acid to produce a full-length protein....
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