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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Bibliografske podrobnosti

Principais autores:	Tobias Bonifert, Irene Gonzalez Menendez, Florian Battke, Yvonne Theurer, Matthis Synofzik, Ludger Schöls, Bernd Wissinger
Format:	Artigo
Jezik:	Inglês
Izdano:	Elsevier 2016-01-01
Serija:	Molecular Therapy: Nucleic Acids
Teme:	antisense oligonucleotides deep intronic mutation OPA1 optic neuropathies splice correction
Online dostop:	http://www.sciencedirect.com/science/article/pii/S2162253117301130
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