Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T > G GAA Splicing Variant in Pompe Disease

Erik Wal, Atze Bergsma, Joon Pijnenburg, Ans van der Ploeg, Pim Pijnappel

Research output: Contribution to journalArticleAcademicpeer-review

51 Citations (Scopus)
22 Downloads (Pure)
Original languageUndefined/Unknown
Pages (from-to)90-100
Number of pages11
JournalMolecular therapy. Nucleic acids
Publication statusPublished - 2017

Research programs

  • EMC MM-01-54-01

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