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CRISPR/Cas9

  • RESEARCH ARTICLE
    CHIP mutations affect the heat shock response differently in human fibroblasts and iPSC-derived neurons
    S. Schuster, E. Heuten, A. Velic, J. Admard, M. Synofzik, S. Ossowski, B. Macek, S. Hauser, L. Schöls
    Disease Models & Mechanisms 2020 13: dmm045096 doi: 10.1242/dmm.045096 Published 12 October 2020

    Summary: Cell viability, expression and immunocytochemical analyses reveal that mutations in STUB1 that cause SCAR16 impair the heat-shock response in patient-derived fibroblasts, but not in iPSC-derived cortical neurons.

  • 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.

  • REVIEW
    Experimental models and tools to tackle glioblastoma
    Faye L. Robertson, Maria-Angeles Marqués-Torrejón, Gillian M. Morrison, Steven M. Pollard
    Disease Models & Mechanisms 2019 12: dmm040386 doi: 10.1242/dmm.040386 Published 6 September 2019

    Summary: This Review discusses preclinical modelling of glioblastoma multiforme to understand its biology and develop therapies, with a focus on mammalian model systems.

  • 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.

  • RESEARCH ARTICLE
    CRISPR/Cas9-mediated homology-directed repair by ssODNs in zebrafish induces complex mutational patterns resulting from genomic integration of repair-template fragments
    Annekatrien Boel, Hanna De Saffel, Wouter Steyaert, Bert Callewaert, Anne De Paepe, Paul J. Coucke, Andy Willaert
    Disease Models & Mechanisms 2018 11: dmm035352 doi: 10.1242/dmm.035352 Published 18 October 2018

    Summary: NGS-based analysis reveals that CRISPR/Cas9-induced double-strand-break repair using single-stranded repair templates is error prone in zebrafish, resulting in complex patterns of integrated repair-template fragments.

  • RESEARCH ARTICLE
    Effective CRISPR/Cas9-based nucleotide editing in zebrafish to model human genetic cardiovascular disorders
    Federico Tessadori, Helen I. Roessler, Sanne M. C. Savelberg, Sonja Chocron, Sarah M. Kamel, Karen J. Duran, Mieke M. van Haelst, Gijs van Haaften, Jeroen Bakkers
    Disease Models & Mechanisms 2018 11: dmm035469 doi: 10.1242/dmm.035469 Published 18 October 2018

    Summary: By using a single-stranded DNA oligonucleotide template in combination with CRISPR/Cas9 in zebrafish, the authors achieved effective germline-transmissible introduction of patient-specific single-nucleotide changes related to cardiovascular disease.

  • RESOURCE ARTICLE
    Apolipoprotein E deficiency accelerates atherosclerosis development in miniature pigs
    Bin Fang, Xueyang Ren, Ying Wang, Ze Li, Lihua Zhao, Manling Zhang, Chu Li, Zhengwei Zhang, Lei Chen, Xiaoxue Li, Jiying Liu, Qiang Xiong, Lining Zhang, Yong Jin, Xiaorui Liu, Lin Li, Hong Wei, Haiyuan Yang, Rongfeng Li, Yifan Dai
    Disease Models & Mechanisms 2018 11: dmm036632 doi: 10.1242/dmm.036632 Published 10 October 2018

    Editor's choice: ApoE knockout pigs displayed severe hypercholesterolemia and spontaneously developed human-like atherosclerotic lesions in the aorta and coronary arteries within 6 months of feeding on a high-fat and high-cholesterol diet.

  • RESEARCH ARTICLE
    A severe atherosclerosis mouse model on the resistant NOD background
    Xugang Wang, Rong Huang, Lichen Zhang, Saichao Li, Jing Luo, Yanrong Gu, Zhijun Chen, Qianqian Zheng, Tianzhu Chao, Wenping Zheng, Xinhui Qi, Li Wang, Yinhang Wen, Yinming Liang, Liaoxun Lu
    Disease Models & Mechanisms 2018 11: dmm033852 doi: 10.1242/dmm.033852 Published 8 October 2018

    Summary: Double knockout of Apoe and Ldlr on the highly atherosclerosis-resistant NOD mouse background results in severe atherosclerosis, which paves the way for the study of severe atherosclerosis in the setting of autoimmunity.

  • RESEARCH ARTICLE
    RNaseT2 knockout rats exhibit hippocampal neuropathology and deficits in memory
    Kerstin W. Sinkevicius, Thomas R. Morrison, Praveen Kulkarni, Martha K. Caffrey Cagliostro, Sade Iriah, Samantha Malmberg, Julia Sabrick, Jennifer A. Honeycutt, Kim L. Askew, Malav Trivedi, Craig F. Ferris
    Disease Models & Mechanisms 2018 11: dmm032631 doi: 10.1242/dmm.032631 Published 27 June 2018

    Summary: The authors characterize a rodent model of RNaseT2 deficiency, which offers insight into the susceptibility of the hippocampus to early inflammation caused by lysosome impairment due to loss of RNaseT2 function.

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