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

作者:Bonifert Tobias; Menendez Irene Gonzalez; Battke Florian; Theurer Yvonne; Synofzik Matthis; Schoels Ludger; Wissinger Bernd
来源:Molecular Therapy - Nucleic Acids, 2016, 5: 13-20.
DOI:10.1038/mtna.2016.93
  • 出版日期2016-11-22