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

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...

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Detalles Bibliográficos
Autores principales: Martinovich, Kelly M., Kicic, Anthony, Stick, Stephen M., Johnsen, Russell D., Fletcher, Sue, Wilton, Steve D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8981082/
https://www.ncbi.nlm.nih.gov/pubmed/35392567
http://dx.doi.org/10.3389/fphar.2022.868863