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    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)

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    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)