Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro an...
Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis
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United States: Public Library of Science
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Language
English
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United States: Public Library of Science
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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 study evaluates eluforsen, a novel, single-strande...
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Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis
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TN_cdi_doaj_primary_oai_doaj_org_article_998648464a6246f1ba96ae9e7ad7986f
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_998648464a6246f1ba96ae9e7ad7986f
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ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0219182
