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Showing 1–6 of 6 results
Advanced filters: Author: Danith H. Ly 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

  • 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

  • This Perspective discusses how the Somatic Cell Genome Editing Consortium aims to accelerate the implementation of safe and effective genome-editing therapies in the clinic.

    • Krishanu Saha
    • Erik J. Sontheimer
    • Jiangbing Zhou
    ReviewsOpen Access
    Nature
    Volume: 592, P: 195-204

  • Peptide nucleic acids (PNAs) can invade canonical nucleic acid dimers but may be limited to certain sequences or perform poorly under physiological conditions. Here PNAs containing Janus bases invade a range of RNA and DNA sequences via Watson-Crick base pairing under near-physiological conditions.

    • Shivaji A. Thadke
    • V. M. Hridya
    • Danith H. Ly
    ResearchOpen Access
    Communications Chemistry
    Volume: 1, P: 1-10

  • Stereo-inverted oligonucleic acids are promising tools for bioengineering but are limited by low availability of suitable monomers. Here the controlled interconversion of helical screw sense in peptide nucleic acid oligomers is achieved through strand displacement.

    • Wei-Che Hsieh
    • Gustavo R. Martinez
    • Danith H. Ly
    ResearchOpen Access
    Communications Chemistry
    Volume: 1, P: 1-10