Article
Antisense oligonucleotide splicing modulation as a novel Cystic Fibrosis therapeutic approach for the W1282X nonsense mutation.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jul 2022
Oren Yifat S, Avizur-Barchad Ofra, Ozeri-Galai Efrat, Elgrabli Renana, Schirelman Meital R, Blinder Tehilla, Stampfer Chava D, Ordan Merav, Laselva Onofrio, Cohen-Cymberknoh Malena, Kerem Eitan, Bear Christine E, Kerem Batsheva
Abstract excerpt
BACKGROUND: Antisense oligonucleotide- based drugs for splicing modulation were recently approved for various genetic diseases with unmet need. Here we aimed to generate skipping over exon 23 of the CFTR transcript, to eliminate the W1282X nonsense mutation and avoid RNA degradation induced by the nonsense mediated mRNA decay mechanism, allowing production of partially active CFTR proteins lacking exon 23....
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