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GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells
Authors: van der Wal, E.; Bergsma, A. J.; van Gestel, T. J. M.; In 't Groen, S. L. M.; Zaehres, H.; Arauzo-Bravo, M. J.; Scholer, H. R.; van der Ploeg, A. T.; Pijnappel, Wwmp
Date of Publication (YYYY-MM-DD): 2017-06-16
Title of Journal: Mol Ther Nucleic Acids
Volume: 7
Start Page: 101
End Page: 115
Document Type: Article
ID: 744092.0