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Showing 1–6 of 6 results
Advanced filters: Author: Rolen M. Quadros Clear advanced filters

  • Mucopolysaccharidosis type I (MPSI) is a lysosomal storage disease caused by insufficient iduronidase (IDUA) activity. Here, the authors use an ex vivo genome editing approach to overexpress IDUA in human hematopoietic stem and progenitor cells and show it can phenotypically correct MSPI in mouse model.

    • Natalia Gomez-Ospina
    • Samantha G. Scharenberg
    • Matthew H. Porteus
    ResearchOpen Access
    Nature Communications
    Volume: 10, P: 1-14

  • Molecular diversity of proprioceptive neuron types (Ia, Ib and II PNs) is unclear. Here, the authors characterized the functional organization and development of eight subtypes of PNs in mice. Importantly, Ia subtypes are plastic, suggesting a role in adaptive proprioception during motor behavior.

    • Haohao Wu
    • Charles Petitpré
    • François Lallemend
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-13

  • In this Perspective, the authors propose genetically engineered mouse models to further the understanding and use of a recently discovered tool, selective endogenous encapsidation for cellular delivery via virus-like particles, in gene delivery and gene therapy applications.

    • Channabasavaiah B. Gurumurthy
    • Rolen M. Quadros
    • Masato Ohtsuka
    Reviews
    Nature Protocols
    Volume: 17, P: 2129-2138

  • A non-viral strategy to introduce large DNA sequences into T cells enables the correction of a pathogenic mutation that causes autoimmunity, and the replacement of an endogenous T-cell receptor with an engineered receptor that can recognize cancer antigens.

    • Theodore L. Roth
    • Cristina Puig-Saus
    • Alexander Marson
    Research
    Nature
    Volume: 559, P: 405-409

  • In this Perspective, the authors discuss strategies for creating a versatile set of 30 genetically engineered mouse models (GEMMs) useful for coronavirus disease 2019 research. In addition, they provide the genetic blueprints needed for developing these GEMMs.

    • Channabasavaiah B. Gurumurthy
    • Rolen M. Quadros
    • Masato Ohtsuka
    Reviews
    Nature Protocols
    Volume: 15, P: 3777-3787