Functional characterization of the common c.-32-13T>G mutation of GAA gene: identification of potential therapeutic agents

Similar Items

• Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset PD patient fibroblasts
  od: Buratti, Emanuele, i dr.
  Izdano: (2020)
• SR protein-mediated inhibition of CFTR exon 9 inclusion: molecular characterization of the intronic splicing silencer
  od: Buratti, Emanuele, i dr.
  Izdano: (2007)
• Functional mapping of the interaction between TDP-43 and hnRNP A2 in vivo
  od: D’Ambrogio, Andrea, i dr.
  Izdano: (2009)
• Splicing mutations in glycogen-storage disease type II: evaluation of the full spectrum of mutations and their relation to patients' phenotypes
  od: Zampieri, Stefania, i dr.
  Izdano: (2011)
• Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease
  od: van der Wal, Erik, i dr.
  Izdano: (2017)