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  1. Article

    Open Access

    Genome-wide detection of CRISPR editing in vivo using GUIDE-tag

    Analysis of off-target editing is an important aspect of the development of safe nuclease-based genome editing therapeutics. in vivo assessment of nuclease off-target activity has primarily been indirect (base...

    Shun-Qing Liang, Pengpeng Liu, Jordan L. Smith, Esther Mintzer… in Nature Communications (2022)

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  2. Article

    Open Access

    Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

    Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but are severely constrained by cargo limits. Simultaneous delivery of multiple vectors can limit dose and e...

    Raed Ibraheim, Phillip W. L. Tai, Aamir Mir, Nida Javeed… in Nature Communications (2021)

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  3. Article

    Open Access

    Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

    Prime editors (PEs) mediate genome modification without utilizing double-stranded DNA breaks or exogenous donor DNA as a template. PEs facilitate nucleotide substitutions or local insertions or deletions withi...

    Pengpeng Liu, Shun-Qing Liang, Chunwei Zheng, Esther Mintzer… in Nature Communications (2021)

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  4. No Access

    Article

    Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

    Efficient Cas9 genome editing in vivo is achieved without viral vectors using chemically modified single-guide RNAs.

    Hao Yin, Chun-Qing Song, Sneha Suresh, Qiongqiong Wu, Stephen Walsh… in Nature Biotechnology (2017)