Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model

• Main Authors: Kelly M. Martinovich (Author), Anthony Kicic (Author), Stephen M. Stick (Author), Russell D. Johnsen (Author), Sue Fletcher (Author), Steve D. Wilton (Author)
• Formato: Livro
• Publicado em: Frontiers Media S.A., 2022-03-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Introduction: Severity and disease progression in people with Cystic Fibrosis (CF) is typically dependent on their genotype. One potential therapeutic strategy for people with specific mutations is exon skipping with antisense oligonucleotides (AO). CFTR exon 9 is an in-frame exon and hence the excl...