Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans-Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa

The promising clinical results obtained for ocular gene therapy in recent years have paved the way for gene supplementation to treat recessively inherited forms of retinal degeneration. The situation is more complex for dominant mutations, as the toxic mutant gene product must be removed. We used sp...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Berger, Adeline, Lorain, Stéphanie, Joséphine, Charlène, Desrosiers, Melissa, Peccate, Cécile, Voit, Thomas, Garcia, Luis, Sahel, José-Alain, Bemelmans, Alexis-Pierre
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2015
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4427870/
https://ncbi.nlm.nih.gov/pubmed/25619725
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.11
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