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Showing 1–5 of 5 results
Advanced filters: Author: Kalin D. Mayberry Clear advanced filters

  • The ability to genetically modify haematopoietic stem cells would allow the durable treatment of a diverse range of genetic disorders but gene delivery to the bone marrow has not been achieved. Here lipid nanoparticles that target and deliver mRNA to 14 unique cells within the bone marrow are presented.

    • Xizhen Lian
    • Sumanta Chatterjee
    • Daniel J. Siegwart
    Research
    Nature Nanotechnology
    Volume: 19, P: 1409-1417

  • A comparison of fetal hemoglobin gene editing strategies using human sickle cell disease donor cells and in vivo transplantation finds that adenine base editing of the –175A>G site in the γ-globin gene promoters results in durable and potent expression.

    • Thiyagaraj Mayuranathan
    • Gregory A. Newby
    • Jonathan S. Yen
    Research
    Nature Genetics
    Volume: 55, P: 1210-1220

  • Detailed mechanistic insight into fetal globin gene induction during hypoxia-associated stress erythropoiesis provides new therapeutic approaches to treat β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia.

    • Ruopeng Feng
    • Thiyagaraj Mayuranathan
    • Mitchell J. Weiss
    Research
    Nature
    Volume: 610, P: 783-790

  • A custom adenine base editor can edit the variant of the β-globin gene that causes sickle cell disease into a non-pathogenic variant in human and mouse cells, and transplantation of the edited cells rescues sickle cell disease in mice.

    • Gregory A. Newby
    • Jonathan S. Yen
    • David R. Liu
    Research
    Nature
    Volume: 595, P: 295-302