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-...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Nucleic Acids
Prif Awduron: Bonifert, Tobias, Gonzalez Menendez, Irene, Battke, Florian, Theurer, Yvonne, Synofzik, Matthis, Schöls, Ludger, Wissinger, Bernd
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2016
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC5155325/
https://ncbi.nlm.nih.gov/pubmed/27874857
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.93
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