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Triplex-forming oligonucleotides bind in the major groove of duplex DNA in a sequence-specific manner through the formation of hydrogen bonds. They can be used to inhibit the expression of particular genes.
Cystic fibrosis is a lethal genetic disorder commonly caused by the F508del mutation which is not amenable to gene therapy. Here, the authors use triplex-forming PNA molecules and donor DNA in biodegradable polymer nanoparticles to correct F508del and achieve clinically relevant levels of gene editing.