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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Main Authors: Tobias Bonifert (Author), Irene Gonzalez Menendez (Author), Florian Battke (Author), Yvonne Theurer (Author), Matthis Synofzik (Author), Ludger Schöls (Author), Bernd Wissinger (Author)
Format: Book
Published: Elsevier, 2016-01-01T00:00:00Z.
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