Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, encoding an anion channel that conducts chloride and bicarbonate across epithelial membranes. Mutations that disrupt pre-mRNA splicing occur in >15% of CF cases....

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Detalles Bibliográficos
Autores principales: Michaels, Wren E, Bridges, Robert J, Hastings, Michelle L
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
RNA and RNA-protein complexes
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7367209/
https://www.ncbi.nlm.nih.gov/pubmed/32520327
http://dx.doi.org/10.1093/nar/gkaa490

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7367209/
https://www.ncbi.nlm.nih.gov/pubmed/32520327
http://dx.doi.org/10.1093/nar/gkaa490

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