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  1. Article ; Online: Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

    Raed Ibraheim / Phillip W. L. Tai / Aamir Mir / Nida Javeed / Jiaming Wang / Tomás C. Rodríguez / Suk Namkung / Samantha Nelson / Eraj Shafiq Khokhar / Esther Mintzer / Stacy Maitland / Zexiang Chen / Yueying Cao / Emmanouela Tsagkaraki / Scot A. Wolfe / Dan Wang / Athma A. Pai / Wen Xue / Guangping Gao /
    Erik J. Sontheimer

    Nature Communications, Vol 12, Iss 1, Pp 1-

    2021  Volume 17

    Abstract: Long-term expression of Cas9 following precision genome editing in vivo may lead to undesirable consequences. Here we show that a single-vector, self-inactivating AAV system containing Cas9 nuclease, guide, and DNA donor can use homology-directed repair ... ...

    Abstract Long-term expression of Cas9 following precision genome editing in vivo may lead to undesirable consequences. Here we show that a single-vector, self-inactivating AAV system containing Cas9 nuclease, guide, and DNA donor can use homology-directed repair to correct disease mutations in vivo.
    Keywords Science ; Q
    Language English
    Publishing date 2021-11-01T00:00:00Z
    Publisher Nature Portfolio
    Document type Article ; Online
    Database BASE - Bielefeld Academic Search Engine (life sciences selection)

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  2. Article ; Online: CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease

    Emmanouela Tsagkaraki / Sarah M. Nicoloro / Tiffany DeSouza / Javier Solivan-Rivera / Anand Desai / Lawrence M. Lifshitz / Yuefei Shen / Mark Kelly / Adilson Guilherme / Felipe Henriques / Nadia Amrani / Raed Ibraheim / Tomas C. Rodriguez / Kevin Luk / Stacy Maitland / Randall H. Friedline / Lauren Tauer / Xiaodi Hu / Jason K. Kim /
    Scot A. Wolfe / Erik J. Sontheimer / Silvia Corvera / Michael P. Czech

    Nature Communications, Vol 12, Iss 1, Pp 1-

    2021  Volume 17

    Abstract: Worldwide pandemics of obesity and diabetes prompt an urgent need for new approaches to their prevention and cure. Here the authors present a CRISPR-based strategy that enhances the therapeutic potential of human adipocytes when implanted in obese mice. ...

    Abstract Worldwide pandemics of obesity and diabetes prompt an urgent need for new approaches to their prevention and cure. Here the authors present a CRISPR-based strategy that enhances the therapeutic potential of human adipocytes when implanted in obese mice.
    Keywords Science ; Q
    Language English
    Publishing date 2021-11-01T00:00:00Z
    Publisher Nature Portfolio
    Document type Article ; Online
    Database BASE - Bielefeld Academic Search Engine (life sciences selection)

    More links

    Kategorien

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