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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Detalhes bibliográficos
Publicado no:PLoS One
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2019
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6599119/
https://ncbi.nlm.nih.gov/pubmed/31251792
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0219182
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