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Showing 1–4 of 4 results
Advanced filters: Author: Jacqueline Robinson-Hamm Clear advanced filters

  • In December 2024, the NIH Office of Autoimmune Disease Research in the Office of Research on Women’s Health (OADR-ORWH) hosted a virtual meeting in the ScienceTALKS series entitled ‘The Cancer Autoimmune Connection: Decoding the Paradox’.

    • Xinrui Li
    • Carmen Ufret-Vincenty
    • Victoria K. Shanmugam
    News & Views
    Nature Immunology
    Volume: 26, P: 342-344

  • Site-specific recombination and CRISPR-Cas9 have been used to generate genetically engineered mouse models of cancer. Here the authors compare sarcomas generated using both systems and see similar genetic and cellular phenotypes, suggesting CRISPR-Cas9 can be used to rapidly generate sarcoma models.

    • Jianguo Huang
    • Mark Chen
    • David G. Kirsch
    ResearchOpen Access
    Nature Communications
    Volume: 8, P: 1-11

  • Genome engineering tools, including targeted gene editing and gene regulation, are becoming available to correct the mutations that cause neuromuscular disorders such as muscular dystrophy, spinal muscular atrophy and myotonic dystrophy. This Review summarizes the genome engineering strategies that are under preclinical evaluation for the treatment of degenerative neuromuscular disorders, focusing on the tools that show the greatest potential for clinical translation.

    • Christopher E. Nelson
    • Jacqueline N. Robinson-Hamm
    • Charles A. Gersbach
    Reviews
    Nature Reviews Neurology
    Volume: 13, P: 647-661

  • In the mdx mouse model of Duchenne muscular dystrophy, single intravenous administration of AAV-CRISPR–Cas9 vectors provides efficient genome editing and restoration of dystrophin expression lasting for one year.

    • Christopher E. Nelson
    • Yaoying Wu
    • Charles A. Gersbach
    Research
    Nature Medicine
    Volume: 25, P: 427-432