GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells

Lignende værker

• GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells
  af: Erik van der Wal, et al.
  Udgivet: (2017-06-01)
• Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease
  af: van der Wal, Erik, et al.
  Udgivet: (2017)
• From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides
  af: Bergsma, Atze J, et al.
  Udgivet: (2016)
• Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies
  af: van der Wal, Erik, et al.
  Udgivet: (2018)
• Extension of the Pompe mutation database by linking disease‐associated variants to clinical severity
  af: Niño, Monica Y., et al.
  Udgivet: (2019)