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GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells.
van der Wal, Erik; Bergsma, Atze J; van Gestel, Tom J M; In 't Groen, Stijn L M; Zaehres, Holm; Araúzo-Bravo, Marcos J; Schöler, Hans R; van der Ploeg, Ans T; Pijnappel, W W M Pim.
Affiliation
  • van der Wal E; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Department of Pediatrics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center, 3015 GE Rotterdam, the
  • Bergsma AJ; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Department of Pediatrics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center, 3015 GE Rotterdam, the
  • van Gestel TJM; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Department of Pediatrics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center, 3015 GE Rotterdam, the
  • In 't Groen SLM; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Department of Pediatrics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center, 3015 GE Rotterdam, the
  • Zaehres H; Department of Cell and Developmental Biology, Max Planck Institute for Molecular Biomedicine, 48149 Münster, Germany.
  • Araúzo-Bravo MJ; Department of Cell and Developmental Biology, Max Planck Institute for Molecular Biomedicine, 48149 Münster, Germany.
  • Schöler HR; Department of Cell and Developmental Biology, Max Planck Institute for Molecular Biomedicine, 48149 Münster, Germany; Westphalian Wilhelms-University, Medical Faculty, 48149 Münster, Germany.
  • van der Ploeg AT; Department of Pediatrics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center, 3015 GE Rotterdam, the Netherlands.
  • Pijnappel WWMP; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Department of Pediatrics, Erasmus Medical Center, 3015 CN Rotterdam, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center, 3015 GE Rotterdam, the
Mol Ther Nucleic Acids ; 7: 101-115, 2017 Jun 16.
Article in En | MEDLINE | ID: mdl-28624186
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2017 Type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2017 Type: Article