splice correctiontherapeutics

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antisense oligonucleotide mediated splice correction of a deep intronic mutation in opa1

;Tobias Bonifert;Irene Gonzalez Menendez;Florian Battke;Yvonne Theurer;Matthis Synofzik;Ludger Schöl...

2016 coordination chemistry reviews DOI: 10.1038/mtna.2016.93 155 views

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antisense oligonucleotides deep intronic mutation opa1 pharmacology