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Showing 1–5 of 5 results
Advanced filters: Author: Elias Quijano Clear advanced filters

  • Gene editing approaches are widely used for correcting mutations, but their application is largely limited to cells and not living animals. Here the authors show that in vivoγPNA-mediated editing of a β-globin mutation is promoted by SCF and leads to sustained normalization of blood haemoglobin levels β-thalassemic mice.

    • Raman Bahal
    • Nicole Ali McNeer
    • Peter M. Glazer
    ResearchOpen Access
    Nature Communications
    Volume: 7, P: 1-14

  • 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.

    • Nicole Ali McNeer
    • Kavitha Anandalingam
    • Marie E. Egan
    Research
    Nature Communications
    Volume: 6, P: 1-11

  • The correction of genetic defects in utero could allow for improved outcomes of gene therapy. Here, the authors demonstrate safe delivery of nanoparticles to fetal mouse tissues, and show that nanoparticles containing peptide nucleic acids to edit the beta-globin gene are effective in a mouse model of beta-thalassemia.

    • Adele S. Ricciardi
    • Raman Bahal
    • W. Mark Saltzman
    ResearchOpen Access
    Nature Communications
    Volume: 9, P: 1-11

  • Borosins are ribosomally encoded and posttranslationally modified peptide (RiPP) natural products featuring amide-backbone α-N-methylation. Here, the authors report the discovery and characterization of type IV borosin ‘split’ pathways encoding distinct, separate α-N-methyltransferases and precursor peptide substrates.

    • Fredarla S. Miller
    • Kathryn K. Crone
    • Michael F. Freeman
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-14