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Genome editing

  • REVIEW
    CRISPR/Cas9-mediated genome editing in nonhuman primates
    Yu Kang, Chu Chu, Fang Wang, Yuyu Niu
    Disease Models & Mechanisms 2019 12: dmm039982 doi: 10.1242/dmm.039982 Published 16 October 2019

    Summary: This Review discusses the history and development of genome editing in non-human primates, as well as the challenges and prospects facing this technology now and in the future.

  • 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.

  • RESEARCH ARTICLE
    Disruption of asxl1 results in myeloproliferative neoplasms in zebrafish
    Evisa Gjini, Chang-Bin Jing, Ashley T. Nguyen, Deepak Reyon, Emma Gans, Michiel Kesarsing, Joshua Peterson, Olga Pozdnyakova, Scott J. Rodig, Marc R. Mansour, Keith Joung, A. Thomas Look
    Disease Models & Mechanisms 2019 12: dmm035790 doi: 10.1242/dmm.035790 Published 7 May 2019

    Summary: Homozygous loss of asxl1 in zebrafish leads to apoptosis of newly formed HSCs by upregulation of bim and bid. Half of the asxl1+/− zebrafish had MPNs by 5 months of age.

  • 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.

  • EDITORIAL
    Spotlight on zebrafish: the next wave of translational research
    E. Elizabeth Patton, David M. Tobin
    Disease Models & Mechanisms 2019 12: dmm039370 doi: 10.1242/dmm.039370 Published 7 March 2019

    Summary: Zebrafish are a highly versatile and relevant organism for human disease modelling. This Editorial highlights the recent zebrafish research published in DMM.

  • EDITORIAL
    From gene to treatment: supporting rare disease translational research through model systems
    Julija Hmeljak, Monica J. Justice (Editor-in-Chief)
    Disease Models & Mechanisms 2019 12: dmm039271 doi: 10.1242/dmm.039271 Published 22 February 2019

    Summary: This Editorial discusses the importance of model systems with accurate face, construct, target and predictive validity for rare disease research.

  • AT A GLANCE
    Generating mouse models for biomedical research: technological advances
    Channabasavaiah B. Gurumurthy, Kevin C. Kent Lloyd
    Disease Models & Mechanisms 2019 12: dmm029462 doi: 10.1242/dmm.029462 Published 8 January 2019

    Summary: Newer molecular technologies to precisely and efficiently manipulate the mammalian genome are enabling the production of more scientifically valuable animal models.

  • RESOURCE ARTICLE
    CRISPR-Cas9 human gene replacement and phenomic characterization in Caenorhabditis elegans to understand the functional conservation of human genes and decipher variants of uncertain significance
    Troy A. McDiarmid, Vinci Au, Aaron D. Loewen, Joseph Liang, Kota Mizumoto, Donald G. Moerman, Catharine H. Rankin
    Disease Models & Mechanisms 2018 11: dmm036517 doi: 10.1242/dmm.036517 Published 26 November 2018

    Summary: Here, we provide a CRISPR-Cas9 human gene replacement and phenomic characterization strategy to directly replace Caenorhabditis elegans genes with their human orthologs for disease variant modeling and therapeutic screening.

  • REVIEW
    Modeling epigenetic modifications in renal development and disease with organoids and genome editing
    Carmen Hurtado del Pozo, Elena Garreta, Juan Carlos Izpisúa Belmonte, Nuria Montserrat
    Disease Models & Mechanisms 2018 11: dmm035048 doi: 10.1242/dmm.035048 Published 20 November 2018

    Summary: In this Review, we provide an overview on how epigenetic processes are altered in kidney development and disease, and discuss how CRISPR-modified kidney organoids can help us to understand the function of epigenetic marks.

  • EDITORIAL
    Zebrafish knock-ins swim into the mainstream
    Sergey V. Prykhozhij, Jason N. Berman
    Disease Models & Mechanisms 2018 11: dmm037515 doi: 10.1242/dmm.037515 Published 24 October 2018

    Summary: With the co-publication of three research articles describing CRISPR/Cas9-based approaches for knocking in mutations in zebrafish (dmm035352, dmm035469 and dmm035972), this Editorial reflects on the current state of genome editing for disease modeling in zebrafish.

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