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Showing 1–8 of 8 results
Advanced filters: Author: Pieter R. Cullis Clear advanced filters

  • Long-circulating, transfection-competent LNP-mRNA systems are key for effective extrahepatic delivery. Here, authors show that LNPs with high bilayer lipid ratios yield high mRNA encapsulation, prolonged circulation, and enhanced transfection in extrahepatic tissues.

    • Miffy Hok Yan Cheng
    • Yao Zhang
    • Pieter R. Cullis
    ResearchOpen Access
    Nature Communications
    Volume: 16, P: 1-12

  • Every organelle in a eukaryotic cell contains enzymes with specialized functions, unique to that organelle. But the endosome does things differently — it is regulated, in part, by a unique lipid called lysobisphosphatidic acid (LBPA). This lipid seems to be crucial, because anti-LBPA antibodies affect both the structure and function of the late endosome. Moreover, LBPA is recognized by autoimmune sera from patients with antiphospholipid syndrome, indicating that it may be involved in pathogenesis of this disease.

    • Sandra L. Schmid
    • Pieter R. Cullis
    News & Views
    Nature
    Volume: 392, P: 135-136

  • The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many nucleic acid-based therapies enabled by nanoparticle delivery.

    • Akin Akinc
    • Martin A. Maier
    • Pieter R. Cullis
    Comments & Opinion
    Nature Nanotechnology
    Volume: 14, P: 1084-1087

  • Efficient siRNA delivery remains a key challenge to realizing the full potential of RNAi-based therapeutics. Semple et al. accomplish unprecedented potency for liposome-mediated siRNA delivery by applying rational design to refine an empirically identified cationic lipid widely regarded as the benchmark for use in lipid nanoparticles.

    • Sean C Semple
    • Akin Akinc
    • Michael J Hope
    Research
    Nature Biotechnology
    Volume: 28, P: 172-176

  • This Review provides an overview of four platform technologies that are currently used in the clinic for delivery of nucleic acid therapeutics, describing their properties, discussing technical advancements that led to clinical approval, and highlighting examples of approved genetic drugs that make use of these technologies.

    • Jayesh A. Kulkarni
    • Dominik Witzigmann
    • Roy van der Meel
    Reviews
    Nature Nanotechnology
    Volume: 16, P: 630-643

  • Tran et al. combine high throughput sequencing, structural biology and computational simulation to determine the HLA allele and antibody-defined epitope frequencies in renal transplant patients and donors. These results demonstrate the feasibility of HLA epitope matching using data from a national transplantation program.

    • Jenny N. Tran
    • Oliver P. Günther
    • Zoltán Kaló
    ResearchOpen Access
    Communications Biology
    Volume: 4, P: 1-14