P.218 - Antisense oligonucleotides promote exon inclusion and correct the common c.-32-13T > G (IVS1) GAA splicing variant in iPS-derived skeletal muscle cells from Pompe patients
| Autor: | Pijnappel, W., van der Wal, E., Bergsma, A., van Gestel, T., Pijnenburg, J., In ‘t Groen, S., Zaehres, H., Araúzo-Bravo, M., Schöler, H., van der Ploeg, A. |
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| Zdroj: | In Neuromuscular Disorders October 2017 27 Supplement 2:S161-S161 |
| Databáze: | ScienceDirect |
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