Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis

Cystic fibrosis (CF) is caused by mutations in the gene encoding the epithelial chloride channel CF transmembrane conductance regulator (CFTR) protein. The most common mutation is a deletion of three nucleotides leading to the loss of phenylalanine at position 508 (p.Phe508del) in the protein. This...

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Detalles Bibliográficos
Autores principales: Beumer, Wouter, Swildens, Jim, Leal, Teresinha, Noel, Sabrina, Anthonijsz, Herma, van der Horst, Geert, Kuiperij-Boersma, Hester, Potman, Marko, van Putten, Charlotte, Biasutto, Patricia, Platenburg, Gerard, de Jonge, Hugo, Henig, Noreen, Ritsema, Tita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6599119/
https://www.ncbi.nlm.nih.gov/pubmed/31251792
http://dx.doi.org/10.1371/journal.pone.0219182

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