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Antisense oligonucleotide splicing modulation as a novel Cystic Fibrosis therapeutic approach for the W1282X nonsense mutation.
Oren YS, Avizur-Barchad O, Ozeri-Galai E, Elgrabli R, Schirelman MR, Blinder T, Stampfer CD, Ordan M, Laselva O, Cohen-Cymberknoh M, Kerem E, Bear CE, Kerem B. Oren YS, et al. Among authors: ozeri galai e. J Cyst Fibros. 2022 Jul;21(4):630-636. doi: 10.1016/j.jcf.2021.12.012. Epub 2021 Dec 28. J Cyst Fibros. 2022. PMID: 34972649 Free article.
Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation.
Oren YS, Irony-Tur Sinai M, Golec A, Barchad-Avitzur O, Mutyam V, Li Y, Hong J, Ozeri-Galai E, Hatton A, Leibson C, Carmel L, Reiter J, Sorscher EJ, Wilton SD, Kerem E, Rowe SM, Sermet-Gaudelus I, Kerem B. Oren YS, et al. Among authors: ozeri galai e. J Cyst Fibros. 2021 Sep;20(5):865-875. doi: 10.1016/j.jcf.2021.06.003. Epub 2021 Jul 2. J Cyst Fibros. 2021. PMID: 34226157 Free PMC article.
The presence of extra chromosomes leads to genomic instability.
Passerini V, Ozeri-Galai E, de Pagter MS, Donnelly N, Schmalbrock S, Kloosterman WP, Kerem B, Storchová Z. Passerini V, et al. Among authors: ozeri galai e. Nat Commun. 2016 Feb 15;7:10754. doi: 10.1038/ncomms10754. Nat Commun. 2016. PMID: 26876972 Free PMC article.