Journal of Cystic Fibrosis

Journal Information
ISSN / EISSN : 1569-1993 / 1873-5010
Current Publisher: Elsevier BV (10.1016)
Total articles ≅ 10,132
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Latest articles in this journal

Michael M. Rey,
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.008

The publisher has not yet granted permission to display this abstract.
, Anika Marand, Sinan Muldur, Alex Hopke, Hui Min Leung, Denis De La Flor, Grace Park, Hanna Pinsky, Lauren B. Guthrie, Guillermo J. Tearney, et al.
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.01.012

The publisher has not yet granted permission to display this abstract.
Rosa Coucke, Audrey Chansard, Véronique Bontemps, Dominique Grenet, Dominique Hubert, Clémence Martin, Elise Lammertyn, Emmanuelle Bardin, Veerle Bulteel, Frédérique Chedevergne, et al.
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.009

The publisher has not yet granted permission to display this abstract.
Lauren M. Castner, Madsen Zimbric, Shannon Cahalan, Corey Powell,
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.001

The publisher has not yet granted permission to display this abstract.
, Kent Green, Lue Philipsen, Rikke Mulvad Sandvik, Marianne Skov, Frederik Buchvald, Tacjana Pressler,
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.004

The publisher has not yet granted permission to display this abstract.
, Nicola J. Ronan, Denver Mooney, Clodagh McGettigan, David Mullane, Muireann NiChroinin, , Desmond M. Murphy, Mairead McCarthy, Yvonne McCarthy, et al.
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2020.12.023

The publisher has not yet granted permission to display this abstract.
Daniel K. Crawford, Jasper Mullenders, Johanna Pott, Sylvia F. Boj, Shira Landskroner-Eiger,
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.01.009

Abstract:
Background Promoting full-length protein production is a requisite step to address some of the remaining unmet medical need for those with Cystic Fibrosis (CF) nonsense alleles. ELX-02 promotes read-through of mRNA transcripts bearing nonsense mutations, including the most common CF nonsense allele G542X, in several different preclinical models including human bronchial epithelial cells. Here we evaluate ELX-02 mediated read-through using the CFTR-dependent Forskolin-induced swelling (FIS) assay across a selection of G542X genotype patient derived organoids (PDOs). Methods CFTR functional restoration was evaluated in ELX-02 treated G542X homozygous and heterozygous PDOs in the CFTR-dependent FIS assay. CFTR mRNA abundance and integrity were evaluated by qPCR and Nanostring analysis while PDO protein was detected by capillary based size-exclusion chromatography. Results PDOs homozygous for G542X or heterozygous with a second minimally functional allele had significantly increased CFTR activity with ELX-02 in a dose-dependent fashion across a variety of forskolin induction concentrations. The functional increases are similar to those obtained with tezacaftor/ivacaftor in F508del homozygous PDOs. Increased CFTR C- and B-band protein was observed in accordance with increased function. In addition, ELX-02 treatment of a G542X/G542X PDO results in a 5-fold increase in CFTR mRNA compared with vehicle treated, resulting in normalization of CFTR mRNA as measured via Nanostring. Conclusions These data with ELX-02 in PDOs are consistent with previous G542X model evaluations. These results also support the on-going clinical evaluation of ELX-02 as a read-through agent for CF caused by the G542X allele.
, Scott H. Donaldson, Carla A. Frederick, Steven D. Freedman, Daniel Gelfond, , Andrea Kelly, Michael R. Narkewicz, Jessica E. Pittman, Felix Ratjen, et al.
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.003

The publisher has not yet granted permission to display this abstract.
Anne Munck, Kevin W Southern, Carlo Castellani, Karin M De Winter-De Groot, Silvia Gartner, , , Sarah J Mayell, Marijke Proesmans, Dorota Sands, et al.
Published: 1 February 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.006

The publisher has not yet granted permission to display this abstract.
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