Open reading frame correction using splice-switching antisense oligonucleotides for the treatment of cystic fibrosis

CFTR gene mutations that result in the introduction of premature termination codons (PTCs) are common in cystic fibrosis (CF). This mutation type causes a severe form of the disease, likely because of low CFTR messenger RNA (mRNA) expression as a result of nonsense-mediated mRNA decay, as well as th...

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Detalles Bibliográficos
Autores principales: Michaels, Wren E., Pena-Rasgado, Cecilia, Kotaria, Rusudan, Bridges, Robert J., Hastings, Michelle L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: National Academy of Sciences 2022
Materias:
Biological Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8784102/
https://www.ncbi.nlm.nih.gov/pubmed/35017302
http://dx.doi.org/10.1073/pnas.2114886119

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8784102/
https://www.ncbi.nlm.nih.gov/pubmed/35017302
http://dx.doi.org/10.1073/pnas.2114886119

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