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

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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Bibliographic Details
Main Authors: Tobias Bonifert, Irene Gonzalez Menendez, Florian Battke, Yvonne Theurer, Matthis Synofzik, Ludger Schöls, Bernd Wissinger
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotides
deep intronic mutation
OPA1
optic neuropathies
splice correction
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301130

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http://www.sciencedirect.com/science/article/pii/S2162253117301130

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