GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells
- van der Wal, E.
- Bergsma, A.J.
- van Gestel, T.J.M.
- in ‘t Groen, S.L.M.
- Zaehres, H.
- Araúzo-Bravo, M.J.
- Schöler, H.R.
- van der Ploeg, A.T.
- Pijnappel, W.W.M.P.
Journal:
Molecular Therapy - Nucleic Acids
ISSN: 2162-2531
Year of publication: 2017
Volume: 7
Pages: 101-115
Type: Article