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Showing 1–12 of 12 results
Advanced filters: Author: Carsten T. Charlesworth Clear advanced filters

  • Pre-existing antibodies against Cas9 proteins represent a potential issue for gene therapies, including those targeting the eye. Here the authors assess the presence of intraocular antibodies, and show that Cas9 antibodies were prevalent in human serum but not the eye, unless prior bacterial infection occurred.

    • Marcus A. Toral
    • Carsten T. Charlesworth
    • Vinit B. Mahajan
    ResearchOpen Access
    Nature Communications
    Volume: 13, P: 1-9

  • In vivo gene editing hinges on identifying an ideal delivery vehicle from numerous candidates. Here, authors establish the GFP-on mouse model capable of translating successful adenine base editing to a fluorescent readout thus enabling the rapid evaluation of genome editing delivery vehicles.

    • Carla Dib
    • Jack A. Queenan
    • Agnieszka D. Czechowicz
    ResearchOpen Access
    Nature Communications
    Volume: 16, P: 1-13

  • A new GWAS of schizophrenia (11,260 cases and 24,542 controls) and meta-analysis identifies 50 new associated loci and 145 loci in total. The common variant association signal is highly enriched in mutation-intolerant genes and in regions under strong background selection.

    • Antonio F. Pardiñas
    • Peter Holmans
    • James T. R. Walters
    Research
    Nature Genetics
    Volume: 50, P: 381-389

  • Shortages of donor blood for transfusions can have severe medical consequences, and ex vivo production of red blood cells offers a potential solution. Here authors developed synthetic EPO receptors, which allow erythropoiesis (red blood cell production) without the need for expensive EPO.

    • Aadit P. Shah
    • Kiran R. Majeti
    • M. Kyle Cromer
    ResearchOpen Access
    Nature Communications
    Volume: 16, P: 1-15

  • A global network of researchers was formed to investigate the role of human genetics in SARS-CoV-2 infection and COVID-19 severity; this paper reports 13 genome-wide significant loci and potentially actionable mechanisms in response to infection.

    • Mari E. K. Niemi
    • Juha Karjalainen
    • Chloe Donohue
    ResearchOpen Access
    Nature
    Volume: 600, P: 472-477

  • Cas9-specific antibodies and reactive T cells are found in the majority of healthy adult human serum samples analyzed. Such preexisting adaptive immunity should be taken into consideration as the CRISPR–Cas9 system moves toward clinical trials.

    • Carsten T. Charlesworth
    • Priyanka S. Deshpande
    • Matthew H. Porteus
    Research
    Nature Medicine
    Volume: 25, P: 249-254

  • To realize the full potential of genetic engineering of haematopoietic stem cells for a broad range of clinical indications, substantial immunological challenges must be overcome. These include innate and adaptive immune responses to gene-therapy reagents and adaptive immune responses to neoantigens expressed by genetically engineered cells.

    • Carsten T. Charlesworth
    • Ian Hsu
    • Hiromitsu Nakauchi
    Reviews
    Nature Reviews Immunology
    Volume: 22, P: 719-733