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  1. No Access

    Article

    Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

    CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold tremendous potential to effectively treat disease-causing mutations with diverse cellular origin. However, it is currently impos...

    Qiang Cheng, Tuo Wei, Lukas Farbiak, Lindsay T. Johnson… in Nature Nanotechnology (2020)

  2. Article

    Disrupting off-target Cas9 activity in the liver

    Off-target genome editing in the liver can be reduced by using lipid nanoparticles to deliver oligonucleotides that disrupt the secondary structure of single-guide RNAs as well as short interfering RNAs target...

    Sean A. Dilliard, Daniel J. Siegwart in Nature Biomedical Engineering (2022)

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  3. Article

    Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs

    Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of medicines that directly reprogramme the central dogma of biology to prevent and treat disease. However, multiple biological barr...

    Sean A. Dilliard, Daniel J. Siegwart in Nature Reviews Materials (2023)

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    Article

    Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells

    Therapeutic genome editing of haematopoietic stem cells (HSCs) would provide long-lasting treatments for multiple diseases. However, the in vivo delivery of genetic medicines to HSCs remains challenging, espec...

    **zhen Lian, Sumanta Chatterjee, Yehui Sun, Sean A. Dilliard… in Nature Nanotechnology (2024)