Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease.
van der Wal E, Bergsma AJ, Pijnenburg JM, van der Ploeg AT, Pijnappel WWMP
Mol Ther Nucleic Acids. 2017 Jun 16;7:90-100. doi: 10.1016/j.omtn.2017.03.001. Epub 2017 Mar 14.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 190694 | Sf-U7snRNA-ESE-AS-NsiI | Basic vector used for the expression of U7-based antisense oligonucleotides |
| 190695 | SF-U7snRNA-CypA-E4 | Control vector expressing a U7-based antisense oligonucleotide targeting CypA exon 4 |