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Exon skipping

  • SPECIAL ARTICLE
    The use of genetically humanized animal models for personalized medicine approaches
    Annemieke Aartsma-Rus, Maaike van Putten
    Disease Models & Mechanisms 2020 13: dmm041673 doi: 10.1242/dmm.041673 Published 1 October 2019

    Summary: Personalized medicine approaches benefit from humanized animal models. Here, we outline the usefulness, caveats and considerations for generating and using these models for pre-clinical studies of Duchenne muscular dystrophy.

  • RESOURCE ARTICLE
    CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene
    Tatiana V. Egorova, Evgenia D. Zotova, Denis A. Reshetov, Anna V. Polikarpova, Svetlana G. Vassilieva, Dmitry V. Vlodavets, Alexey A. Gavrilov, Sergey V. Ulianov, Vladimir L. Buchman, Alexei V. Deykin
    Disease Models & Mechanisms 2019 12: dmm037655 doi: 10.1242/dmm.037655 Published 25 April 2019

    Summary: The authors describe the creation of a mouse strain that reproduces a newly identified deletion mutation in a DMD patient in Russia, and present the characteristics of this new model.

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