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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Detalles Bibliográficos
Autores principales: Berger, Adeline, Lorain, Stéphanie, Joséphine, Charlène, Desrosiers, Melissa, Peccate, Cécile, Voit, Thomas, Garcia, Luis, Sahel, José-Alain, Bemelmans, Alexis-Pierre
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4427870/
https://www.ncbi.nlm.nih.gov/pubmed/25619725
http://dx.doi.org/10.1038/mt.2015.11

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