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Exon-skipping antisense oligonucleotides for cystic fibrosis therapy

Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (CF), and the CFTR-W1282X nonsense mutation causes a severe form of CF. Although Trikafta and other CFTR-modulation therapies benefit most CF patients, targeted therapy for patients with the W1282X...

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Detalles Bibliográficos
Autores principales:	Kim, Young Jin, Sivetz, Nicole, Layne, Jessica, Voss, Dillon M., Yang, Lucia, Zhang, Qian, Krainer, Adrian R.
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/PMC8784140/ https://www.ncbi.nlm.nih.gov/pubmed/35017301 http://dx.doi.org/10.1073/pnas.2114858118

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