Antisense Oligonucleotide Mediated Splice Correction of a Deep Intronic Mutation in OPA1

Inherited optic neuropathies (ION) present an important cause of blindness in the European working-age population. Recently we reported the discovery of four independent families with deep intronic mutations in the main inherited optic neuropathies gene OPA1. These deep intronic mutations cause mis-...

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Detalles Bibliográficos
Autores principales: Bonifert, Tobias, Gonzalez Menendez, Irene, Battke, Florian, Theurer, Yvonne, Synofzik, Matthis, Schöls, Ludger, Wissinger, Bernd
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5155325/
https://www.ncbi.nlm.nih.gov/pubmed/27874857
http://dx.doi.org/10.1038/mtna.2016.93

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